H. Kiem

H. Deeg, J. Radich, S. Heimfeld, 2006, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Robust Expansion of HIV CAR T Cells Following Antigen Boosting in ART-Suppressed Nonhuman Primates.

Mark R. Enstrom, C. Fennessey, B. Keele, 2020, Blood.

Efficient generation, purification, and expansion of CD34(+) hematopoietic progenitor cells from nonhuman primate-induced pluripotent stem cells.

H. Kiem, S. D’Souza, Jennifer L. Gori, 2012, Blood.

Pharmacologically regulated in vivo selection in a large animal.

C. von Kalle, A. Gown, T. Clackson, 2002, Blood.

Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons

L. Naldini, P. Horn, H. Kiem, 2002, Gene Therapy.

Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations

H. Kiem, Meei-Li W Huang, K. Jerome, 2021, Molecular therapy. Methods & clinical development.

American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells.

K. Cornetta, M. Sadelain, C. A. Blau, 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

Comparative analysis of risk factors for acute graft-versus-host disease and for chronic graft-versus-host disease according to National Institutes of Health consensus criteria.

R. Storb, B. Storer, F. Appelbaum, 2011, Blood.

Duration of immunosuppressive treatment for chronic graft-versus-host disease.

R. Storb, B. Storer, F. Appelbaum, 2004, Blood.

The use of granulocyte colony-stimulating factor during retroviral transduction on fibronectin fragment CH-296 enhances gene transfer into hematopoietic repopulating cells in dogs.

R. Storb, P. Mcsweeney, H. Kiem, 1999, Blood.

Efficient marking of murine long-term repopulating stem cells targeting unseparated marrow cells at low lentiviral vector particle concentration.

H. Kiem, P. Kurre, M. Harkey, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

Engineering resistance to CD33-targeted immunotherapy in normal hematopoiesis by CRISPR/Cas9-deletion of CD33 exon 2

H. Kiem, R. Walter, S. Sichel, 2018, Leukemia.

Highly efficient gene transfer into baboon marrow repopulating cells using GALV-pseudotype oncoretroviral vectors produced by human packaging cells.

S. Riddell, P. Horn, H. Kiem, 2002, Blood.

A capsid-modified, conditionally replicating oncolytic adenovirus vector expressing TRAIL Leads to enhanced cancer cell killing in human glioblastoma models.

H. Kiem, A. Lieber, B. Beard, 2007, Cancer research.

Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

S. Heimfeld, H. Kiem, G. Trobridge, 2016, Nature Communications.

Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS

M. Kamata, J. Zack, H. Kiem, 2017, PLoS pathogens.

A Cure for HIV Infection: “Not in My Lifetime” or “Just Around the Corner”?

B. Walker, M. Lederman, D. Kuritzkes, 2016, Pathogens & immunity.

Immune inactivation of anti-simian immunodeficiency virus chimeric antigen receptor T cells in rhesus macaques

L. Picker, M. Roederer, Y. Fukazawa, 2021, Molecular therapy. Methods & clinical development.

Efficient Enrichment of Gene-Modified Primary T Cells via CCR5-Targeted Integration of Mutant Dihydrofolate Reductase

D. Rawlings, H. Kiem, C. Peterson, 2018, Molecular therapy. Methods & clinical development.

Proliferation-Linked Apoptosis of Adoptively Transferred T Cells after IL-15 Administration in Macaques

S. Riddell, H. Kiem, T. Gooley, 2013, PloS one.

Foamy viral vector integration sites in SCID-repopulating cells after MGMTP140K-mediated in vivo selection

D. Rawlings, H. Kiem, G. Trobridge, 2015, Gene Therapy.

Extended core sequences from the cHS4 insulator are necessary for protecting retroviral vectors from silencing position effects.

G. Stamatoyannopoulos, G. Felsenfeld, H. Kiem, 2007, Human gene therapy.

Effects of HOXB4 Overexpression on Ex Vivo Expansion and Immortalization of Hematopoietic Cells from Different Species

R. Humphries, J. Schwartz, H. Kiem, 2007, Stem cells.

Differential Effects of HOXB4 on Nonhuman Primate Short- and Long-Term Repopulating Cells

B. Storer, R. Humphries, H. Kiem, 2006, PLoS medicine.

Use of a SCID mouse/human lymphoma model to evaluate cytokine-induced killer cells with potent antitumor cell activity

I. Weissman, I. Schmidt-Wolf, H. Kiem, 1991, The Journal of experimental medicine.

Refining Current Scientific Priorities and Identifying New Scientific Gaps in HIV-Related Heart, Lung, Blood, and Sleep Research.

P. Ridker, M. Engelgau, S. Glynn, 2017, AIDS research and human retroviruses.

Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model.

David A. Williams, C. von Kalle, T. Neff, 2003, The Journal of clinical investigation.

Induction of cytotoxic T-lymphocyte responses to enhanced green and yellow fluorescent proteins after myeloablative conditioning.

S. Riddell, H. Kiem, J. Storek, 2004, Blood.

Overexpression of glutathione-S-transferase, MGSTII, confers resistance to busulfan and melphalan.

H. Kiem, J. Slattery, M. Harkey, 2005, Cancer investigation.

In-Vivo Gene Therapy with Foamy Virus Vectors

H. Kiem, Olivier Humbert, Y. Rajawat, 2019, Viruses.

Retrovirus-mediated gene transduction into canine peripheral blood repopulating cells.

C. von Kalle, H. Deeg, F. Appelbaum, 1994, Blood.

Overexpression of Glutathione-S-Transferase, MGSTII, Confers Resistance to Busulfan and Melphalan

H. Kiem, J. Slattery, M. Harkey, 2005 .

Human CD34+ cells do not express glutathione S-transferases alpha

H. Kiem, J. Slattery, M. Czerwiński, 1997, Gene Therapy.

Lentivirus-mediated Gene Transfer in Hematopoietic Stem Cells Is Impaired in SHIV-infected, ART-treated Nonhuman Primates.

Philip R. Gafken, Hannah W. Miller, Shiu-Lok Hu, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Retrovirus-mediated transfer of the cDNA for human glucocerebrosidase into peripheral blood repopulating cells of patients with Gaucher's disease.

T. Reynolds, R. Storb, A. Miller, 1997, Human gene therapy.

Human hematopoietic stem cell maintenance and myeloid cell development in next-generation humanized mouse models.

A. Rongvaux, H. Kiem, S. Radtke, 2019, Blood advances.

Insights into leukemia-initiating cell frequency and self-renewal from a novel canine model of leukemia.

R. Humphries, H. Kiem, Xiao-Bing Zhang, 2011, Experimental hematology.

In Vivo Hematopoietic Stem Cell Transduction.

T. Papayannopoulou, H. Kiem, A. Lieber, 2017, Hematology/oncology clinics of North America.

Improved short‐term engraftment of lentivirally versus gammaretrovirally transduced allogeneic canine repopulating cells

T. Neff, H. Kiem, S. Gerull, 2007, The journal of gene medicine.

Distinct hematopoietic stem/progenitor cell populations are responsible for repopulating NOD/SCID mice compared with nonhuman primates.

B. Wood, P. Horn, H. Kiem, 2003, Blood.

Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor.

J. Rasko, S. Heyward, A. Miller, 1998, Blood.

Immuno-Therapy with Anti-CTLA4 Antibodies in Tolerized and Non-Tolerized Mouse Tumor Models

H. Kiem, S. Roffler, A. Lieber, 2011, PloS one.

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

I. Bernstein, A. Scharenberg, D. Rawlings, 2018, Molecular therapy. Methods & clinical development.

Differential effects of bexarotene on intrinsic and extrinsic pathways in TRAIL-induced apoptosis in two myeloid leukemia cell lines

H. Deeg, J. Pagel, D. Hockenbery, 2007, Leukemia & lymphoma.

Hematopoietic Stem Cell Behavior in Non-human Primates

Abkowitz, P. Guttorp, P. Lansdorp, 2007 .

Erratum: Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors.

Z. Izsvák, Hongjie Wang, T. Papayannopoulou, 2021, Molecular therapy. Methods & clinical development.

Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol.

T. Neff, P. Horn, H. Kiem, 2004, Blood.

Gene Transfer into Marrow Repopulating Cells: Comparison Between Amphotropic and Gibbon Ape Leukemia Virus Pseudotyped Retroviral Vectors in a Competitive Repopulation Assay in Baboons

James M. Allen, S. Heyward, A. Miller, 1997 .

Gene transfer into marrow repopulating cells: comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons.

S. Heyward, A. Miller, H. Kiem, 1997, Blood.

Oral abstracts of the 21st International AIDS Conference 18–22 July 2016, Durban, South Africa

Michael I. Jordan, Donald A. Jackson, J. G. Ndirangu, 2016, Journal of the International AIDS Society.

Allogeneic hematopoietic stem cell transplantation for myelofibrosis.

H. Deeg, J. Radich, R. Storb, 2003, Blood.

Gene therapy studies in a canine model of X-linked severe combined immunodeficiency.

P. Felsburg, H. Kiem, B. Sorrentino, 2015, Human gene therapy. Clinical development.

Intravenous injection of a foamy virus vector to correct canine SCID-X1.

T. Torgerson, A. Scharenberg, D. Rawlings, 2014, Blood.

In vivo Selection of Autologous MGMT Gene-Modified Cells Following Reduced Intensity Conditioning with BCNU and Temozolomide in the Dog Model

H. Kiem, Jennifer L. Gori, Garyfalia Karponi, 2012, Cancer Gene Therapy.

Long-term Regulation of Genetically Modified Primary Hematopoietic Cells in Dogs.

C. von Kalle, C. A. Blau, H. Kiem, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Stable marking and transgene expression without progression to monoclonality in canine long-term hematopoietic repopulating cells transduced with lentiviral vectors.

H. Kiem, G. Trobridge, K. Keyser, 2010, Human gene therapy.

Long-term polyclonal and multilineage engraftment of methylguanine methyltransferase P140K gene-modified dog hematopoietic cells in primary and secondary recipients.

T. Neff, H. Kiem, G. Trobridge, 2009, Blood.

High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector.

B. Wood, R. Humphries, R. Humphries, 2008, The Journal of clinical investigation.

Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia.

C. von Kalle, A. Nienhuis, C. A. Blau, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene Transfer in Adeno-Associated Virus Seropositive Rhesus Macaques Following Rapamycin Treatment and Subcutaneous Delivery of AAV6, but Not Retargeted AAV6 Vectors

Shiu-Lok Hu, H. Kiem, Meei-Li W Huang, 2021 .

Myelosuppressive conditioning improves autologous engraftment of genetically marked hematopoietic repopulating cells in dogs.

C. von Kalle, R. Storb, H. Kiem, 1995, Blood.

Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies

P. Dexheimer, P. Malik, H. Kiem, 2014, Molecular therapy. Methods & clinical development.

Cyclosporine inhibits the development of green fluorescent protein (GFP)-specific immune responses after transplantation of GFP-expressing hematopoietic repopulating cells in dogs.

H. Kiem, X. Zhang, J. Morris, 2005, Human gene therapy.

Envelope-Specific Adaptive Immunity following Transplantation of Hematopoietic Stem Cells Modified with VSV-G Lentivirus

H. Kiem, C. Peterson, P. Becker, 2020, Molecular therapy. Methods & clinical development.

Retrovirus-Mediated Gene Transduction Into Canine Peripheral Blood Repopulating Cells

H. Deeg, R. Storb, F. Appelbaum, 1994 .

Toward a stem cell gene therapy for breast cancer.

S. Tuve, H. Kiem, N. Kiviat, 2009, Blood.

Towards a stem cell gene therapy for breast cancer

S. Tuve, H. Kiem, N. Kiviat, 2009 .

Gene transfer into baboon repopulating cells: A comparison of Flt-3 Ligand and megakaryocyte growth and development factor versus IL-3 during ex vivo transduction.

P. Horn, H. Kiem, P. Kurre, 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

Comparison of Mesenchymal Stem Cells from Different Tissues to Suppress T-Cell Activation

H. Kiem, K. Keyser, K. Beagles, 2007, Cell transplantation.

Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy.

T. Torgerson, A. Scharenberg, D. Rawlings, 2018, Blood advances.

Large Animal Models for Foamy Virus Vector Gene Therapy

P. Horn, H. Kiem, G. Trobridge, 2012, Viruses.

Devouring the Hematopoietic Stem Cell: Setting the Table for Marrow Cell Transplantation.

H. Kiem, M. Cowan, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

GaLV pseudotyped vectors and cationic lipids transduce human CD34+ cells.

H. Kiem, C. Lynch, P. Anklesaria, 1997, Human gene therapy.

Immunosuppression After Marrow Transplant CTLA4Ig, and 100 cGy Total Body Irradiation Before and Pharmacologic Stable Mixed Hematopoietic Chimerism in Dogs Given Donor Antigen,

H. Deeg, R. Storb, P. Mcsweeney, 2013 .

Targeted gene disruption to cure HIV

H. Kiem, K. Jerome, D. Stone, 2013, Current opinion in HIV and AIDS.

Host variation in type I interferon signaling genes (MX1), CCR5Δ32, and MHC class I alleles in treated HIV+ non-controllers predict viral reservoir size

F. Hecht, D. Kroetz, E. Wan, 2022, bioRxiv.

Transgene expression by a large fraction of dendritic cells following autologous transplantation of retrovirally transduced CD34 cells.

H. Kiem, J. Storek, 2006, Stem cells and development.

e- Blood Efﬁcient generation, puriﬁcation, and expansion of CD34 (cid:1) hematopoietic progenitor cells from nonhuman primate–induced pluripotent stem cells

H. Kiem, Jennifer L. Gori, J. Adair, 2012 .

progenitor cells from nonhuman primate induced pluripotent stem cells hematopoietic + Efficient generation, purification, and expansion of CD34

H. Kiem, L. Sunita, Jennifer L. Gori, 2012 .

Psoralen and ultraviolet A irradiation (PUVA) as therapy for steroid-resistant cutaneous acute graft-versus-host disease.

R. Storb, W. Leisenring, F. Appelbaum, 2002, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

ASSESSMENT, AND STREAMLINED PREPARATION OF LOW CYTOTOXICITY LENTIVIRAL VECTORS FOR MOBILIZED HUMAN HEMATOPOIETIC STEM CELL TRANSDUCTION.

D. Wojchowski, H. Kiem, P. Toran, 2020, Experimental hematology.

A retrospective comparison of tacrolimus versus cyclosporine with methotrexate for immunosuppression after allogeneic hematopoietic cell transplantation with mobilized blood cells.

R. Storb, B. Storer, F. Appelbaum, 2011, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Evaluation of NIH consensus criteria for classification of late acute and chronic GVHD.

B. Storer, F. Appelbaum, E. Warren, 2009, Blood.

Current and Future Preparative Regimens for Bone Marrow Transplantation in Thalassemia a

H. Deeg, J Wagner, K. Sullivan, 1998, Annals of the New York Academy of Sciences.

Combination of HOXB4 and Delta-1 ligand improves expansion of cord blood cells.

I. Bernstein, R. Humphries, H. Kiem, 2010, Blood.

Isolation of a Highly Purified HSC-enriched CD34+CD90+CD45RA− Cell Subset for Allogeneic Transplantation in the Nonhuman Primate Large-animal Model

H. Kiem, Lucrezia Colonna, L. Kean, 2020, Transplantation direct.

Breast cancer resistance protein (BCRP/ABCG2) does not confer resistance to gemtuzumab ozogamicin and calicheamicin-γ1 in acute myeloid leukemia cells

I. Bernstein, H. Kiem, R. Walter, 2004, Leukemia.

Evaluation of biodistribution and safety of adenovirus vectors containing group B fibers after intravenous injection into baboons.

A. Gaggar, H. Kiem, S. Ni, 2005, Human gene therapy.

Canine Models for Transplantation and Gene Therapy

R. Storb, H. Kiem, 2000 .

MISTRG mice support engraftment and assessment of nonhuman primate hematopoietic stem and progenitor cells.

A. Rongvaux, H. Kiem, S. Radtke, 2019, Experimental hematology.

Expansion and transduction of nonenriched human cord blood cells using HS-5 conditioned medium and FLT3-L.

S. Heimfeld, H. Kiem, B. Torok-Storb, 2000, Journal of hematotherapy & stem cell research.

Safeguarding nonhuman primate iPS cells with suicide genes.

H. Kiem, K. L. Watts, Jennifer L. Gori, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy for Fanconi anemia: one step closer to the clinic.

J. Tolar, J. Bueren, H. Hanenberg, 2012, Human Gene Therapy.

Evaluation of Posttransplant Methotrexate to Facilitate Engraftment in the Canine Major Histocompatibility Complex-Haploidentical Nonmyeloablative Transplant Model

R. Storb, B. Sandmaier, M. Thakar, 2010, Transplantation.

Effective Multi-lineage Engraftment in a Mouse Model of Fanconi Anemia Using Non-genotoxic Antibody-Based Conditioning

Anthony E. Boitano, M. Cooke, D. Scadden, 2020, Molecular therapy. Methods & clinical development.

Minimal conditioning in Fanconi anemia promotes multi‐lineage marrow engraftment at 10‐fold lower cell doses

H. Kiem, Kevin G. Haworth, J. Adair, 2018, The journal of gene medicine.

Cyclophosphamide promotes engraftment of gene-modified cells in a mouse model of Fanconi anemia without causing cytogenetic abnormalities

H. Kiem, G. Trobridge, P. Becker, 2012, Journal of Molecular Medicine.

Intraosseous delivery of platelet-targeted factor VIII lentiviral vector in humanized NBSGW mice

D. Rawlings, H. Kiem, C. Miao, 2022, Blood advances.

The CD33 splice isoform lacking exon 2 as therapeutic target in human acute myeloid leukemia

B. Wood, C. Correnti, H. Kiem, 2020, Leukemia.

Transmission and expansion of HOXB4-induced leukemia in two immunosuppressed dogs: implications for a new canine leukemia model.

B. Sandmaier, M. Thakar, H. Kiem, 2009, Experimental hematology.

Purified canine CD34+Lin- marrow cells transduced with retroviral vectors give rise to long-term multi-lineage hematopoiesis.

R. Storb, P. Mcsweeney, R. Nash, 2001, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Gene transfer into fetal baboon hematopoietic progenitor cells.

H. Kiem, L. Shields, R. Andrews, 1999, Human gene therapy.

Failure of recombinant stem cell factor to enhance engraftment of L-leucyl-L-leucine methyl ester treated canine marrow after irradiation.

H. Deeg, R. Storb, W. Leisenring, 1996, Blood.

Efficient Transduction by an Amphotropic Retrovirus Vector Is Dependent on High-Level Expression of the Cell Surface Virus Receptor

S. Heyward, A. Miller, H. Kiem, 1999, Journal of Virology.

chronic GVHD Evaluation of NIH consensus criteria for classification of late acute and

B. Storer, F. Appelbaum, E. Warren, 2009 .

Treatment change as a predictor of outcome among patients with classic chronic graft-versus-host disease.

B. Storer, F. Appelbaum, E. Warren, 2008, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Allogeneic hematopoietic stem cell transplantation for myeloﬁbrosis

H. Deeg, J. Radich, R. Storb, 2003 .

Chimeric antigen receptor T-cell approaches to HIV cure

H. Kiem, C. Peterson, A. Kuhlmann, 2018, Current opinion in HIV and AIDS.

Combinatorial anti-HIV gene therapy: using a multipronged approach to reach beyond HAART

H. Kiem, C. Peterson, K. Jerome, 2013, Gene Therapy.

Chemoprotection in glioblastoma therapy: reality or a dream?

M. Mrugala, H. Kiem, J. Adair, 2012, CNS oncology.

Sirolimus in combination with cyclosporine or tacrolimus plus methotrexate for prevention of graft-versus-host disease following hematopoietic cell transplantation from unrelated donors.

H. Deeg, R. Storb, F. Appelbaum, 2008, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies

Gregory A. Newby, Hongjie Wang, H. Kiem, 2022, JCI insight.

Hematopoietic stem cell engraftment: a direct comparison between intramarrow and intravenous injection in nonhuman primates.

B. Storer, T. Neff, C. Jung, 2007, Experimental Hematology.

Survival of the Fittest

T. Neff, H. Kiem, Tobias Neff, 1997 .

Nonmyeloablative Hematopoietic Cell Transplantation

C. Flowers, R. Storb, B. Sandmaier, 2001, Current opinion in oncology.

Marrow grafts from HLA-identical siblings for severe aplastic anemia: does limiting the number of transplanted marrow cells reduce the risk of chronic GVHD?

S. Heimfeld, P. Hari, R. Storb, 2016, Bone Marrow Transplantation.

R. Storb, H. Kiem, J. Sanders, 2007, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Morbidity and mortality of chronic GVHD after hematopoietic stem cell transplantation from HLA-identical siblings for patients with aplastic or refractory anemias.

K. Sullivan, R. Storb, H. Kiem, 2002, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Efficient gene transfer in primitive CD34+/CD38lo human bone marrow cells reselected after long-term exposure to GALV-pseudotyped retroviral vector.

C. von Kalle, V. Diehl, R. Storb, 1997, Human gene therapy.

In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors.

Z. Izsvák, W. Uckert, Kamola Saydaminova, 2016, Blood.

Concurrent activation of c-myc and inactivation of bcl-2 by chromosomal translocation in a lymphoblastic lymphoma cell line.

M. Cleary, J. Nourse, H. Kiem, 1990, Oncogene.

In vivo protection of activated Tyr22‐dihydrofolate reductase gene‐modified canine T lymphocytes from methotrexate

H. Kiem, Jennifer L. Gori, B. Beard, 2013, The journal of gene medicine.

Differences in F36VMpl-based in vivo selection among large animal models.

S. Heimfeld, T. Clackson, H. V. von Recum, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells.

H. Kiem, D. Kohn, P. Kurre, 2003, Blood.

Modeling promising nonmyeloablative conditioning regimens in nonhuman primates.

T. Papayannopoulou, H. Kiem, B. Nakamoto, 2014, Human gene therapy.

Coupling endonucleases with DNA end–processing enzymes to drive gene disruption

Sarah K. Baxter, Michael T. Certo, A. Lambert, 2012, Nature Methods.

HLA-Haploidentical Hematopoietic Cell Transplantation for Treatment of Non-Malignant Diseases Using Nonmyeloablative Conditioning and Post-Transplant Cyclophosphamide.

T. Torgerson, R. Storb, B. Sandmaier, 2020, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

David A. Williams, C. von Kalle, J. Wagner, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Transmission of Chagas disease via blood transfusions in 2 immunosuppressed pigtailed macaques (Macaca nemestrina).

H. Kiem, A. Baldessari, L. Kuller, 2014, Comparative medicine.

Treatment of Fanconi anemia patients using fludarabine and low-dose total body irradiation followed by unrelated donor hematopoietic cell transplantation

R. Storb, B. Sandmaier, W. Leisenring, 2010, Bone Marrow Transplantation.

TRAILshort Protects against CD4 T Cell Death during Acute HIV Infection

S. Lewin, G. Bren, A. Badley, 2019, The Journal of Immunology.

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector

D. Liggitt, A. Nienhuis, D. Rawlings, 2016, Molecular therapy. Methods & clinical development.

Hepatitis-associated aplastic anemia.

R. Storb, H. Kiem, G. McDonald, 1997, The New England journal of medicine.

Use of the polymerase chain reaction to monitor the effectiveness of ex vivo tumor cell purging.

M. Cleary, I. Schmidt-Wolf, H. Kiem, 1991, Blood.

Ex vivo selection for oncoretrovirally transduced green fluorescent protein-expressing CD34-enriched cells increases short-term engraftment of transduced cells in baboons.

J. Rasko, H. Kiem, P. Kurre, 2002, Human gene therapy.

Factors Associated with Response, CAR-T Cell In Vivo Expansion, and Progression-Free Survival after Repeat Infusions of CD19 CAR-T Cells

D. Maloney, C. Turtle, S. Riddell, 2019, Blood.

transplantation in monkeys Interleukin-7 improves CD4 T cell reconstitution after autologous CD34 cell

D. Maloney, A. Joseph, Hailing Lu, 2013 .

Interleukin-7 improves CD4 T-cell reconstitution after autologous CD34 cell transplantation in monkeys.

D. Maloney, A. Joseph, Hailing Lu, 2003, Blood.

Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells.

Daniel G. Miller, R. Kaul, J. Fletcher, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Efficient Characterization of Retro‐, Lenti‐, and Foamyvector‐Transduced Cell Populations by High‐Accuracy Insertion Site Sequencing

S. Karlsson, H. Glimm, M. Wissler, 2003, Annals of the New York Academy of Sciences.

Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotype oncoretrovirus vectors.

J. Rasko, R. Storb, P. Horn, 2001, Blood.

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

H. Kiem, R. Shahbazi, Kevin G. Haworth, 2019, Nature Materials.

Genetically modified hematopoietic stem cell transplantation for HIV-1-infected patients: can we achieve a cure?

H. Kiem, P. Younan, J. Kowalski, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS

H. Kiem, P. Younan, J. Kowalski, 2013, Viruses.

Screening Clinical Cell Products for Replication Competent Retrovirus: The National Gene Vector Biorepository Experience

K. Cornetta, S. Rosenberg, M. Sadelain, 2018, Molecular therapy. Methods & clinical development.

High-throughput genomic mapping of vector integration sites in gene therapy studies.

H. Kiem, G. Trobridge, J. Adair, 2014, Methods in molecular biology.

Current translational and clinical practices in hematopoietic cell and gene therapy.

H. Kiem, D. Digiusto, 2012, Cytotherapy.

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.

Hung Fan, Alessandro Aiuti, Jacqueline Corrigan-Curay, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

Extended Survival of Glioblastoma Patients After Chemoprotective HSC Gene Therapy

D. Silbergeld, M. Mrugala, T. Neff, 2012, Science Translational Medicine.

Novel reporter systems for facile evaluation of I-SceI-mediated genome editing

Nina M. Muñoz, A. Scharenberg, B. Ryu, 2011, Nucleic acids research.

Gene Therapy Extended Survival of Glioblastoma Patients After Chemoprotective HSC

D. Silbergeld, M. Mrugala, T. Neff, 2012 .

Myelodysplasia in 2 Pig-Tailed Macaques (Macaca nemestrina) Associated With Retroviral Vector–Mediated Insertional Mutagenesis and Overexpression of HOXB4

H. Kiem, R. Hukkanen, S. Kelley, 2011, Veterinary pathology.

Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates.

H. Kiem, G. Trobridge, J. McCune, 2010, The Journal of clinical investigation.

Foamy and lentiviral vectors transduce canine long-term repopulating cells at similar efficiency.

James M. Allen, D. Russell, H. Kiem, 2009, Human gene therapy.

Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectors.

J. Fletcher, P. Malik, H. Kiem, 2008, Blood.

cells with HIV-based lentiviral vectors Efficient transduction of pigtailed macaque hematopoietic repopulating

J. Fletcher, P. Malik, H. Kiem, 2008 .

Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus.

Daniel G. Miller, R. Kaul, H. Kiem, 2007, Human gene therapy.

Polyclonal long-term repopulating stem cell clones in a primate model.

C. von Kalle, M. Wissler, J. Tisdale, 2002, Blood.

Myeloablative irradiation in non‐human primates

H. Kiem, K. L. Watts, B. Wood, 2009, Journal of medical primatology.

SARS-CoV-2 Receptor ACE2 Is an Interferon-Stimulated Gene in Human Airway Epithelial Cells and Is Detected in Specific Cell Subsets across Tissues

Fabian J Theis, Alison Yu, Alex K. Shalek, 2020, Cell.

Interleukin-7 improves reconstitution of antiviral CD4 T cells.

T. Kalina, D. Maloney, D. Liggitt, 2005, Clinical immunology.

Human Embryonic Stem Cell-Derived Cardiomyocytes Regenerate Non-Human Primate Hearts

Charles E. Murry, Lil Pabon, Hans-Peter Kiem, 2014, Nature.

Factors associated with durable EFS in adult B-cell ALL patients achieving MRD-negative CR after CD19 CAR T-cell therapy.

D. Maloney, C. Turtle, S. Riddell, 2019, Blood.

CAR T-cell therapy for cancer and HIV through novel approaches to HIV-associated haematological malignancies.

H. Kiem, T. Uldrick, Blake J. Rust, 2020, The Lancet. Haematology.

The response to lymphodepletion impacts PFS in patients with aggressive non-Hodgkin lymphoma treated with CD19 CAR T cells.

D. Maloney, C. Turtle, S. Riddell, 2019, Blood.

CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

H. Kiem, Olivier Humbert, C. Samuelson, 2020, British journal of haematology.

Improved gene transfer into canine hematopoietic repopulating cells using CD34-enriched marrow cells in combination with a gibbon ape leukemia virus–pseudotype retroviral vector

R. Storb, A. Miller, P. Mcsweeney, 1999, Gene Therapy.

Protection of Stem Cell-Derived Lymphocytes in a Primate AIDS Gene Therapy Model after In Vivo Selection

Nina M. Muñoz, John J. Rossi, Hans-Peter Kiem, 2009, PloS one.

Hematopoietic stem cell transduction and amplification in large animal models.

Hans-Peter Kiem, H. Kiem, G. Trobridge, 2005, Human gene therapy.

Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy.

T. Neff, H. Kiem, B. Beard, 2005, Blood.

Stem Cell Selection In Vivo Using Foamy Vectors Cures Canine Pyruvate Kinase Deficiency

P. Malik, H. Kiem, R. A. Wu, 2012, PloS one.

Simian varicella virus in pigtailed macaques (Macaca nemestrina): clinical, pathologic, and virologic features.

H. D. Liggitt, R. Grant, H. Kiem, 2009, Comparative medicine.

Cell and Gene Therapy for HIV Cure.

H. Kiem, C. Peterson, Christopher W. Peterson, 2018, Current topics in microbiology and immunology.

Efficient gene transfer to hematopoietic repopulating cells using concentrated RD114-pseudotype vectors produced by human packaging cells.

T. Neff, P. Horn, H. Kiem, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

Direct comparison of steady-state marrow, primed marrow, and mobilized peripheral blood for transduction of hematopoietic stem cells in dogs.

H. Kiem, Jesse Thompson, L. Peterson, 2003, Human gene therapy.

Efficient Generation of Antigen-Specific Cytotoxic T Cells Using Retrovirally Transduced CD40-Activated B Cells1

M. Tanimoto, Y. Obata, M. Harada, 2002, The Journal of Immunology.

Transduced CD40-Activated B Cells Cytotoxic T Cells Using Retrovirally Efficient Generation of Antigen-Specific

M. Tanimoto, Y. Obata, M. Harada, 2002 .

International AIDS Society global scientific strategy: towards an HIV cure 2016

Richard Jefferys, David Margolis, Jintanat Ananworanich, 2016, Nature Medicine.

Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells.

O. Elemento, S. Rafii, Michael G. Poulos, 2015, The Journal of clinical investigation.

De novo generation of CD4 T cells against viruses present in the host during immune reconstitution.

T. Kalina, D. Maloney, D. Dittmer, 2005, Blood.

De novo generation of T cells against viruses present in the body during immune reconstitution

T. Kalina, Hailing Lu, H. Kiem, 2004 .

Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes.

D. Boger, B. Torbett, B. Ryu, 2019, Blood.

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells.

B. Torbett, D. Rawlings, H. Kiem, 2014, Blood.

AMD3100 re-dosing fails to repeatedly mobilize hematopoietic stem cells in the non-human primate and humanized mouse.

H. Kiem, S. Radtke, Olivier Humbert, 2020, Experimental hematology.

Gene Therapy for Fanconi Anemia in Seattle: Clinical Experience and Next Steps

H. Kiem, P. Becker, J. Adair, 2016 .

Efficient generation of nonhuman primate induced pluripotent stem cells.

H. Kiem, G. Trobridge, Xiao-Bing Zhang, 2011, Stem cells and development.

A novel murine retrovirus identified during testing for helper virus in human gene transfer trials

T. Reynolds, G. Wolgamot, A. Miller, 1996, Journal of virology.

Host variation in type I interferon signaling genes (MX1), C–C chemokine receptor type 5 gene, and major histocompatibility complex class I alleles in treated HIV+ noncontrollers predict viral reservoir size

F. Hecht, D. Kroetz, E. Wan, 2022, AIDS.

Autologous, Gene-Modified Hematopoietic Stem and Progenitor Cells Repopulate the Central Nervous System with Distinct Clonal Variants

H. Kiem, J. Estes, C. Peterson, 2019, Stem cell reports.

Hematopoietic stem-cell behavior in nonhuman primates.

Peter Guttorp, Bryan E Shepherd, Hans-Peter Kiem, 2007, Blood.

Myeloid-biased HSC require Semaphorin 4A from the bone marrow niche for self-renewal under stress and life-long persistence

B. Göttgens, N. Wilson, P. Kharchenko, 2021, bioRxiv.

Engraftment of DLA-haploidentical marrow with ex vivo expanded, retrovirally transduced cytotoxic T lymphocytes.

J. Thompson, R. Storb, B. Sandmaier, 2001, Blood.

CCR5-edited gene therapies for HIV cure: Closing the door to viral entry.

H. Kiem, C. Peterson, Kevin G. Haworth, 2017, Cytotherapy.

Foamy virus vector integration sites in normal human cells

Hans-Peter Kiem, Daniel G. Miller, R. Kaul, 2006, Proceedings of the National Academy of Sciences of the United States of America.

Foamy-virus-mediated gene transfer to canine repopulating cells.

D. Russell, H. Kiem, G. Trobridge, 2007, Blood.

Rapid 1-hour transduction of whole bone marrow leads to long-term repopulation of murine recipients with lentivirus-modified hematopoietic stem cells

H. Kiem, P. Kurre, P. Anandakumar, 2006, Gene Therapy.

Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A

H. Kiem, P. Becker, Kevin G. Haworth, 2018, Haematologica.

Cyclophosphamide promotes engraftment of gene-modified cells in a mouse model of Fanconi anemia without causing cytogenetic abnormalities

H. Kiem, G. Trobridge, P. Becker, 2012, Journal of Molecular Medicine.

Cocal-pseudotyped lentiviral vectors resist inactivation by human serum and efficiently transduce primate hematopoietic repopulating cells.

H. Kiem, R. A. Wu, G. Trobridge, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Lentiviral vector conferring resistance to mycophenolate mofetil and sensitivity to ganciclovir for in vivo T-cell selection

H. Kiem, M. Jensen, R. Nash, 2007, Gene Therapy.

A retroviral vector common integration site between leupaxin and zinc finger protein 91 (ZFP91) observed in baboon hematopoietic repopulating cells.

H. Kiem, G. Trobridge, B. Beard, 2010, Experimental hematology.

Clonal kinetics and single-cell transcriptional profiling of CAR-T cells in patients undergoing CD19 CAR-T immunotherapy

Mark R. Enstrom, P. Linsley, R. Gottardo, 2020, Nature Communications.

Canine models of gene-modified hematopoiesis.

H. Kiem, B. Beard, 2009, Methods in molecular biology.

Humoral immune response limits gene therapy in canine MPS I [letter]

C. Lutzko, H. Kiem, I. Dubé, 1996 .

Humoral immune response limits gene therapy in canine MPS I.

C. Lutzko, H. Kiem, I. Dubé, 1996, Blood.

Synthetic introns enable splicing factor mutation-dependent targeting of cancer cells

O. Abdel-Wahab, R. Bradley, H. Kiem, 2022, Nature Biotechnology.

Rethinking the Regulatory Infrastructure for Human Gene Transfer Clinical Trials.

A. Levine, C. June, H. Kiem, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Mouse Models in Hematopoietic Stem Cell Gene Therapy and Genome Editing.

H. Kiem, S. Radtke, Olivier Humbert, 2019, Biochemical pharmacology.

Survival of the fittest : in vivo selection and stem cell gene therapy

Hans-Peter Kiem, T. Neff, H. Kiem, 2005 .

Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia.

M. Thakar, H. Kiem, M. Eapen, 2017, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Hematopoietic Stem Cell Approaches to Cancer.

H. Kiem, J. Adair, Sara P. Kubek, 2017, Hematology/oncology clinics of North America.

Outcome of allogeneic hematopoietic cell transplantation from HLA-identical siblings for severe aplastic anemia in patients over 40 years of age.

R. Storb, B. Sandmaier, W. Leisenring, 2010, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

A phase I/II study of mycophenolate mofetil in combination with cyclosporine for prophylaxis of acute graft-versus-host disease after myeloablative conditioning and allogeneic hematopoietic cell transplantation.

H. Deeg, R. Storb, B. Storer, 2005, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome.

H. Deeg, J. Radich, R. Storb, 2002, Blood.

Highly efficient gene transfer into preterm CD34 hematopoietic progenitor cells.

H. Kiem, L. Shields, R. Andrews, 2000, American journal of obstetrics and gynecology.

A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates

Mark R. Enstrom, H. Kiem, S. Radtke, 2017, Science Translational Medicine.

Charting a clear path: the ASGCT Standardized Pathways Conference.

David A. Williams, F. Bushman, T. Flotte, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Hematopoietic-stem-cell-based gene therapy for HIV disease.

Hans-Peter Kiem, J. McCune, S. Deeks, 2012, Cell stem cell.

and Development Factor Interleukin-6, Stem Cell Factor, FLT-3 Ligand, and Megakaryocyte Growth Recombinant Human Fibronectin Fragment CH-296 in Combination With Improved Gene Transfer Into Baboon Marrow Repopulating Cells Using

S. Heyward, A. Miller, H. Kiem, 2013 .

Reduced Toxicity and Prompt Engraftment After Minimal Conditioning of a Patient With Fanconi Anemia Undergoing Hematopoietic Stem Cell Transplantation From an HLA-Matched Unrelated Donor

B. Sandmaier, H. Kiem, M. Pulsipher, 2003, Journal of pediatric hematology/oncology.

A Gut Reaction to SIV and SHIV Infection: Lower Dysregulation of Mucosal T Cells during Acute Infection Is Associated with Greater Viral Suppression during cART

J. Mullins, D. Fuller, H. Kiem, 2021, Viruses.

Blockade of alpha6-integrin reveals diversity in homing patterns among human, baboon, and murine cells.

T. Papayannopoulou, H. Kiem, H. Bonig, 2009, Stem cells and development.

No evidence of clonal dominance after transplant of HOXB4-expanded cord blood cells in a nonhuman primate model.

B. Wood, R. Humphries, H. Kiem, 2014, Experimental hematology.

Host Variation in Interferon, MHC Class I, Glycosylation, and Viral Transcription Genes Predict HIV Persistence

M. Carrington, F. Hecht, D. Kroetz, 2021 .

HIV infection results in clonal expansions containing integrations within pathogenesis-related biological pathways.

H. Kiem, Kevin G. Haworth, J. Adair, 2018, JCI insight.

Gene therapy enhances chemotherapy tolerance and efficacy in glioblastoma patients.

D. Silbergeld, D. Born, K. Swanson, 2014, The Journal of clinical investigation.

Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy

Mark R. Enstrom, H. Kiem, S. Radtke, 2020, Molecular therapy. Methods & clinical development.

Sort-purification of human CD34+CD90+ cells reduces target cell population and improves lentiviral transduction

Mark R. Enstrom, H. Kiem, S. Radtke, 2019, bioRxiv.

Factors Impacting Progression-Free Survival after CD19-Specific CAR-T Cell Therapy for Relapsed/Refractory Aggressive B-Cell Non-Hodgkin Lymphoma

D. Maloney, C. Turtle, S. Riddell, 2018, Blood.

Research priorities for an HIV cure: International AIDS Society Global Scientific Strategy 2021

J. Nabukenya, T. Ndung’u, S. Lewin, 2021, Nature Medicine.

Enhancing infection-resistant cells for HIV cure in the non-human primate model

H. Kiem, J. Estes, C. Peterson, 2017 .

Lessons from London and Berlin: Designing A Scalable Gene Therapy Approach for HIV Cure.

H. Kiem, C. Peterson, Christopher W. Peterson, 2019, Cell stem cell.

Stem cell-derived CAR T cells traffic to HIV reservoirs in macaques.

J. Zack, H. Kiem, I. Chen, 2021, JCI insight.

Evidence for persistence of the SHIV reservoir early after MHC haploidentical hematopoietic stem cell transplantation

S. Furlan, Shiu-Lok Hu, H. Kiem, 2018, Nature Communications.

Lack of viral control and development of cART escape mutations in macaques after bone marrow transplantation

Roger E Bumgarner, J. Mullins, H. Kiem, 2015 .

Loss of immune homeostasis dictates SHIV rebound after stem-cell transplantation.

R. Sékaly, Shiu-Lok Hu, Clarisse Benne, 2017, JCI insight.

Safety and Efficacy of Combination Antiretroviral Therapy in Human Immunodeficiency Virus-Infected Adults Undergoing Autologous or Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancies.

J. Schiffer, H. Kiem, C. Johnston, 2016, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Efficacy and Toxicity of JCAR014 in Combination with Durvalumab for the Treatment of Patients with Relapsed/Refractory Aggressive B-Cell Non-Hodgkin Lymphoma

D. Maloney, C. Turtle, S. Riddell, 2018, Blood.

Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells

Mark R. Enstrom, H. Kiem, C. Peterson, 2021, Molecular therapy. Methods & clinical development.

Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy

A. Reik, M. Holmes, Shiu-Lok Hu, 2018, PLoS pathogens.

The frequency of multipotent CD133(+)CD45RA(-)CD34(+) hematopoietic stem cells is not increased in fetal liver compared with adult stem cell sources.

H. Kiem, S. Radtke, Kevin G. Haworth, 2016, Experimental hematology.

All-trans retinoic acid facilitates oncoretrovirus-mediated transduction of hematopoietic repopulating stem cells.

I. Bernstein, S. Collins, H. Kiem, 2001, Journal of hematotherapy & stem cell research.

Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy

Gregory A. Newby, M. Weiss, H. Kiem, 2023, bioRxiv.

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates.

E. Rebar, P. Gregory, M. Holmes, 2016, Blood.

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

A. Scharenberg, D. Rawlings, Ciaran M. Lee, 2019, Science Translational Medicine.

(211)Astatine-Conjugated Monoclonal CD45 Antibody-Based Nonmyeloablative Conditioning for Stem Cell Gene Therapy.

B. Sandmaier, H. Kiem, D. Hamlin, 2015, Human gene therapy.

In vivo gene therapy for canine SCID-X1 using cocal-pseudotyped lentiviral vector.

Mark R. Enstrom, H. Kiem, S. Radtke, 2020, Human gene therapy.

Abrogated cryptic activation of lentiviral transfer vectors

H. Kiem, R. Luche, Joerg Enssle, 2012, Scientific Reports.

NOD/SCID repopulating cells contribute only to short-term repopulation in the baboon

H. Kiem, B. Beard, P. Mezquita, 2008, Gene Therapy.

Longitudinal assessment of morbidity and acute graft-versus-host disease after allogeneic hematopoietic cell transplantation: retrospective analysis of a multicenter phase III study.

R. Storb, W. Leisenring, F. Appelbaum, 2009, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

T-cell immunotherapy: looking forward.

D. Baltimore, A. Ribas, S. Rosenberg, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Factors associated with outcomes after a second CD19-targeted CAR T-cell infusion for refractory B cell malignancies.

M. Bar, D. Maloney, C. Turtle, 2020, Blood.

Bringing gene therapy to where it's needed.

H. Kiem, S. Radtke, 2022, Trends in molecular medicine.

Induction of transgene-specific cytotoxic T lymphocyte responses after transplantation of gene-modified CD34+ cells despite nonablative immunosuppressive conditioning.

S. Riddell, H. Kiem, B. Beard, 2008, Human gene therapy.

Third Generation CD20 Targeted CAR T-Cell Therapy (MB-106) for Treatment of Patients with Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma

M. Bar, D. Maloney, E. Warren, 2020 .

High Efficacy and Low Toxicity of MB-106, a Third Generation CD20 Targeted CAR-T for Treatment of Relapsed/Refractory B-NHL and CLL

D. Maloney, E. Warren, C. Turtle, 2022, Transplantation and Cellular Therapy.

Predictors of Cytopenia after Treatment with Axicabtagene Ciloleucel in Patients with Large Cell Lymphoma

D. Maloney, C. Turtle, H. Kiem, 2020 .

In Vivo Murine-Matured Human CD3+ Cells as a Preclinical Model for T Cell-Based Immunotherapies

H. Kiem, Kevin G. Haworth, J. Adair, 2017, Molecular therapy. Methods & clinical development.

T-Cell Immunotherapy: Looking Forward: T Cell Immunotherapy: Optimizing Trial Design Bethesda, Maryland 10-11 September 2013.

D. Baltimore, A. Ribas, S. Rosenberg, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

T Cell Immunotherapy: Optimizing Trial Design

D. Baltimore, A. Ribas, S. Rosenberg, 2014 .

transduced cytotoxic T lymphocytes Engraftment of DLA-haploidentical marrow with ex vivo expanded, retrovirally

R. Storb, B. Sandmaier, J. Zaucha, 2013 .

Robust suppression of env‐SHIV viremia in Macaca nemestrina by 3‐drug ART is independent of timing of initiation during chronic infection

Hannah W. Miller, Shiu-Lok Hu, H. Kiem, 2013, Journal of medical primatology.

Envelope fusion protein binding studies in an inducible model of retrovirus receptor expression and in CD34+ cells emphasize limited transduction at low receptor levels

A. Miller, H. Kiem, P. Kurre, 2001, Gene Therapy.

Targeting the Membrane-Proximal C2-Set Domain of CD33 for Improved CD33-Directed Immunotherapy

C. Turtle, C. Correnti, H. Kiem, 2021, Leukemia.

Next-Generation Conditioning for Bone Marrow Transplantation: Paving the Way for CAR-T Cell-Based Conditioning.

H. Kiem, Kevin G. Haworth, 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

Effect of recombinant canine stem cell factor, a c-kit ligand, on hematopoietic recovery after DLA-identical littermate marrow transplants in dogs.

C. von Kalle, H. Deeg, M. Pepe, 1997, Experimental hematology.

Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia

R. Storb, B. Sandmaier, M. Thakar, 2016, Bone Marrow Transplantation.

In vivo selection and chemoprotection after drug resistance gene therapy in a nonmyeloablative allogeneic transplantation setting in dogs.

T. Neff, H. Kiem, S. Gerull, 2007, Human gene therapy.

The effect of imatinib on cytomegalovirus reactivation in hematopoietic cell transplantation.

M. Boeckh, L. Corey, H. Kiem, 2009, Clinical infectious diseases : an official publication of the Infectious Diseases Society of America.

Differential effects of HOXB4 and NUP98-HOXA10hd on hematopoietic repopulating cells in a nonhuman primate model.

R. Humphries, H. Kiem, G. Trobridge, 2011, Human gene therapy.

Outside the Box—Novel Therapeutic Strategies for Glioblastoma

M. Mrugala, H. Kiem, J. Adair, 2012, Cancer journal.

Endothelial Cells Promote Expansion of Long‐Term Engrafting Marrow Hematopoietic Stem and Progenitor Cells in Primates

S. Rafii, Michael G. Poulos, Michael Ginsberg, 2016, Stem cells translational medicine.

DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis

Mark R. Enstrom, M. Porteus, H. Kiem, 2020, Molecular therapy. Methods & clinical development.

VISA - Vector Integration Site Analysis server: a web-based server to rapidly identify retroviral integration sites from next-generation sequencing

Brian Beard, Jonah Hocum, Logan Battrell, 2015, BMC Bioinformatics.

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice.

Z. Izsvák, John C. Davis, T. Papayannopoulou, 2021, Blood advances.

Fanconi anemia type C-deficient hematopoietic cells are resistant to TRAIL (TNF-related apoptosis-inducing ligand)-induced cleavage of pro-caspase-8.

H. Deeg, J. Radich, H. Kiem, 2004, Experimental hematology.

A curative regimen would decrease HIV prevalence but not HIV incidence unless targeted to an ART-naïve population

J. Schiffer, H. Kiem, C. Johnston, 2016, Scientific Reports.

Comparison of Efficacy and Toxicity of CD19-Specific Chimeric Antigen Receptor T-Cells Alone or in Combination with Ibrutinib for Relapsed and/or Refractory CLL

D. Maloney, C. Turtle, S. Riddell, 2018, Blood.

Expansion of SCID repopulating cells does not prove expansion of hematopoietic stem cells.

P. Horn, H. Kiem, 2006, Blood.

Efficient transduction and engraftment of G-CSF-mobilized peripheral blood CD34+ cells in nonhuman primates using GALV-pseudotyped gammaretroviral vectors.

H. Kiem, Julia C. Morris, B. Beard, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Development of Third-generation Cocal Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T-cells.

Thomas M. Schmitt, H. Kiem, G. Trobridge, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Relapsed or Refractory CLL after CD19-Specific CAR-T Therapy: Treatment Patterns and Clinical Outcomes

D. Maloney, C. Turtle, H. Kiem, 2019, Blood.

Ectopic expression of HOXC6 blocks myeloid differentiation and predisposes to malignant transformation.

D. Heckl, H. Kiem, Xiao-Bing Zhang, 2014, Experimental hematology.

Efficient ex vivo expansion of conserved element vaccine-specific CD8+ T-cells from SHIV-infected, ART-suppressed nonhuman primates

J. Mullins, G. Shaw, C. Bollard, 2023, Frontiers in Immunology.

In vivo administration of interferon gamma does not cause marrow aplasia in mice with a targeted disruption of FANCC.

M. Grompe, H. Kiem, P. Kurre, 2002, Experimental hematology.

In vivo HSC prime editing rescues sickle cell disease in a mouse model

Gregory A. Newby, Hongjie Wang, H. Kiem, 2023, Blood.

In vivo HSC transduction in rhesus macaques with an HDAd5/3+ vector targeting desmoglein 2 and transiently overexpressing cxcr4

Hongjie Wang, H. Kiem, A. Lieber, 2022, Blood advances.

Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells.

H. Kiem, S. Radtke, Olivier Humbert, 2022, Molecular therapy : the journal of the American Society of Gene Therapy.

HOXB 4-Expanded Cord Blood Cells in a Nonhuman Primate Model

B. Wood, R. Humphries, H. Kiem, 2014 .

CD34+ Expansion With Delta-1 and HOXB4 Promotes Rapid Engraftment and Transfusion Independence in a Macaca nemestrina Cord Blood Transplant Model.

R. Humphries, H. Kiem, G. Trobridge, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Hematopoietic stem cell expansion facilitates multilineage engraftment in a nonhuman primate cord blood transplantation model.

B. Wood, R. Humphries, H. Kiem, 2012, Experimental hematology.

Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development

I. Weissman, D. Weissman, M. Porteus, 2021 .

Continuous Electrical Oscillations Emerge from a Coupled Network: A Study of the Inferior Olive using Lentiviral Knockdown of Connexin36

John P Welsh, Hans-Peter Kiem, J. Welsh, 2006, The Journal of Neuroscience.

Fundamental role of inferior olive connexin 36 in muscle coherence during tremor.

John P Welsh, Hans-Peter Kiem, J. Welsh, 2004, Proceedings of the National Academy of Sciences of the United States of America.

HIV eradication—from Berlin to Boston

H. Kiem, D. Kohn, P. Cannon, 2014, Nature Biotechnology.

Positive selection of mC46-expressing CD4+ T cells and maintenance of virus specific immunity in a primate AIDS model.

Shiu-Lok Hu, H. Kiem, G. Trobridge, 2013, Blood.

Evidence for persistence of the SHIV reservoir early after MHC haploidentical hematopoietic stem cell transplantation

S. Furlan, Shiu-Lok Hu, H. Kiem, 2018, Nature Communications.

Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers

B. Stoddard, A. Lambert, H. Kiem, 2017, Nature Communications.

A multiplexed barcode approach to simultaneously evaluate gene delivery by adeno-associated virus capsid variants in nonhuman primates

H. Büning, H. Kiem, N. Meumann, 2023, Hepatology communications.

Sorting Out the Best: Enriching Hematopoietic Stem Cells for Gene Therapy and Editing.

H. Kiem, S. Radtke, Olivier Humbert, 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

VISA - Vector Integration Site Analysis server: a web-based server to rapidly identify retroviral integration sites from next-generation sequencing

Daniel G. Miller, H. Kiem, G. Trobridge, 2015, BMC Bioinformatics.

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector

J. Wagner, A. Shimamura, H. Kiem, 2010, Gene Therapy.

Prevalence of hepatitis G virus in patients with aplastic anemia.

R. Storb, W. Leisenring, H. Kiem, 1997, Blood.

Marrow transplantation for hepatitis-associated aplastic anemia: a follow-up of long-term survivors.

H. Deeg, K. Sullivan, R. Storb, 1996, Biology of Blood and Marrow Transplantation.

Tightly regulated gene expression in human hematopoietic stem cells after transduction with helper-dependent Ad5/35 vectors.

H. Cao, H. Kiem, A. Lieber, 2008, Experimental hematology.

Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia.

J. Bueren, H. Kiem, C. D. Heredia, 2017, Current gene therapy.

Concurrent Blockade of α4‐Integrin and CXCR4 in Hematopoietic Stem/Progenitor Cell Mobilization

T. Papayannopoulou, H. Kiem, K. L. Watts, 2009, Stem cells.

Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors

Z. Izsvák, Hongjie Wang, T. Papayannopoulou, 2021, Molecular therapy. Methods & clinical development.

A Combined In Vivo HSC Transduction/Selection Approach Results in Efficient and Stable Gene Expression in Peripheral Blood Cells in Mice

Z. Izsvák, S. Nilsson, Hongjie Wang, 2017, Molecular therapy. Methods & clinical development.

Correction: Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS

M. Kamata, J. Zack, H. Kiem, 2018, PLoS Pathogens.

Gene therapy in the transplantation of allogeneic organs and stem cells.

P. Horn, H. Kiem, C. Figueiredo, 2007, Current Gene Therapy.

Development of a clonality assay for canine hematopoietic stem cells.

E. Ostrander, R. Storb, P. Mcsweeney, 1995, Blood.

Nanoparticle Biokinetics in Mice and Nonhuman Primates.

Richard A. Revia, Forrest M Kievit, Hans-Peter Kiem, 2017, ACS nano.

In Vivo HSC Gene Therapy for Hemoglobinopathies: A Proof of Concept Evaluation in Rhesus Macaques

Hongjie Wang, T. Papayannopoulou, H. Kiem, 2020 .

Immunotherapy with T-Cells Engineered with a Chimeric Antigen Receptor Bearing a Human CD19-Binding Single Chain Variable Fragment for Relapsed or Refractory Acute Lymphoblastic Leukemia and B-Cell Non-Hodgkin Lymphoma

D. Maloney, C. Turtle, S. Riddell, 2018, Blood.

Genetically modified CD34+ cells do not contribute to the mesenchymal compartment after autologous transplantation in the baboon.

H. Kiem, K. Keyser, J. Morris, 2005, Cytotherapy.

Gene therapy and bone marrow transplantation

C. von Kalle, R. Storb, H. Kiem, 1995, Current opinion in oncology.

In Vivo Hematopoietic Stem Cell Gene Therapy for SARS-CoV2 Infection Using a Decoy Receptor

D. Fuller, Hongjie Wang, H. Frizzell, 2022, Human gene therapy.

Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-tumor effects.

J. Radich, J Wagner, R. Storb, 2001, Blood.

Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow transplantation.

H. Deeg, J Wagner, R. Storb, 1997, Blood.

Human Embryonic Stem Cell-Derived Cardiomyocytes Regenerate Non-Human Primate Hearts

C. Don, E. Gill, H. Kiem, 2014, Nature.

CD45-targeted antibody-drug-conjugate successfully conditions for allogeneic hematopoietic stem cell transplantation.

J. Wagner, M. MacMillan, H. Kiem, 2022, Blood.

Severe canine hereditary hemolytic anemia treated by nonmyeloablative marrow transplantation.

R. Storb, J. Zaucha, H. Kiem, 2001, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Differential Effects of HOXB4 on Nonhuman Primate Short- and Long-Term Repopulating Cells

B. Storer, R. Humphries, H. Kiem, 2006, PLoS medicine.

The evolution of viral integration site analysis.

H. Kiem, S. Radtke, 2020, Blood.

Hematopoietic recovery after transplantation is primarily derived from the stochastic contribution of hematopoietic stem cells

Mark R. Enstrom, H. Kiem, S. Radtke, 2021, bioRxiv.

THE EFFECT OF IMATINIB ON CMV REACTIVATION IN HEMATOPOIETIC CELL TRANSPLANTATION

L. Corey, H. Kiem, S. Pergam, 2009 .

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34+ Cells

F. Bushman, M. Mrugala, H. Kiem, 2011 .

Current translational and clinical practices in hematopoietic cell and gene therapy.

H. Kiem, D. Digiusto, 2012, Cytotherapy.

In Vivo Hematopoietic Stem Cell Transduction.

T. Papayannopoulou, H. Kiem, A. Lieber, 2017, Hematology/oncology clinics of North America.

Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-tumor effects.

J Wagner, R. Storb, B. Sandmaier, 2001, Blood.

Efficient manufacturing and engraftment of CCR5 gene-edited HSPCs following busulfan conditioning in nonhuman primates

J. Murray, H. Kiem, M. Carrillo, 2023, Molecular therapy. Methods & clinical development.

Treatment change as a predictor of outcome among patients with classic chronic graft-versus-host disease.

B. Storer, F. Appelbaum, E. Warren, 2008, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome.

H. Deeg, J. Radich, R. Storb, 2002, Blood.

Combinatorial hematopoietic stem cell transplantation and vaccination reduces viral pathogenesis following SHIV89.6P-challenge

H. Kiem, P. Younan, P. Polacino, 2015, Gene Therapy.

Lentivirus-mediated Gene Transfer in Hematopoietic Stem Cells Is Impaired in SHIV-infected, ART-treated Nonhuman Primates.

Philip R. Gafken, Hannah W. Miller, Shiu-Lok Hu, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

A capsid-modified, conditionally replicating oncolytic adenovirus vector expressing TRAIL Leads to enhanced cancer cell killing in human glioblastoma models.

H. Kiem, A. Lieber, B. Beard, 2007, Cancer research.

Factors associated with long-term outcomes of CD19 CAR T-cell therapy for relapsed/refractory CLL

D. Maloney, C. Turtle, H. Kiem, 2023, Blood advances.

Hematopoietic stem-cell behavior in nonhuman primates.

P. Guttorp, J. Abkowitz, P. Lansdorp, 2007, Blood.