Efficacy of lentivirus‐mediated gene therapy in an Omenn syndrome recombination‐activating gene 2 mouse model is not hindered by inflammation and immune dysregulation
暂无分享,去创建一个
L. Notarangelo | K. Dobbs | G. Wagemaker | Valentina Capo | M. C. Castiello | A. Villa | P. Uva | E. Draghici | E. Fontana | M. Bosticardo | Rosita Rigoni | B. Cassani | Lucia Sergi Sergi | Paola Carrera | N. P. van Til | L. Poliani | M. Zanussi | S. Penna | Nicolò Sacchetti | Niek P. van Til
[1] F. Rieux-Laucat,et al. Autoimmune and inflammatory manifestations occur frequently in patients with primary immunodeficiencies , 2017, The Journal of allergy and clinical immunology.
[2] M. Porteus,et al. The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation , 2017, Current opinion in hematology.
[3] David A. Williams,et al. Evolving Gene Therapy in Primary Immunodeficiency. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.
[4] Claudio Mussolino,et al. Refining strategies to translate genome editing to the clinic , 2017, Nature Medicine.
[5] C. Cancrini,et al. Late‐onset combined immune deficiency due to LIGIV mutations in a 12‐year‐old patient , 2017, Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology.
[6] D. Kohn,et al. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing. , 2017, The Journal of allergy and clinical immunology.
[7] L. Notarangelo,et al. Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Severe Combined Immunodeficiency Patients: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects afte , 2017, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.
[8] Pachai Natarajan,et al. CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease , 2017, Science Translational Medicine.
[9] E. Husebye,et al. Autoimmune regulator and self‐tolerance – molecular and clinical aspects , 2016, Immunological reviews.
[10] L. Notarangelo,et al. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency , 2016, Science Translational Medicine.
[11] A. Thrasher,et al. Treating Immunodeficiency through HSC Gene Therapy. , 2016, Trends in molecular medicine.
[12] L. Notarangelo,et al. Human RAG mutations: biochemistry and clinical implications , 2016, Nature Reviews Immunology.
[13] F. Candotti. Advances of gene therapy for primary immunodeficiencies , 2016, F1000Research.
[14] G. Pesole,et al. Intestinal microbiota sustains inflammation and autoimmunity induced by hypomorphic RAG defects , 2016, The Journal of experimental medicine.
[15] S. Hacein-Bey-Abina,et al. Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand? , 2016, Human gene therapy.
[16] T. Kodama,et al. Fezf2 Orchestrates a Thymic Program of Self-Antigen Expression for Immune Tolerance , 2015, Cell.
[17] J. B. Oliveira,et al. Broad-spectrum antibodies against self-antigens and cytokines in RAG deficiency. , 2015, The Journal of clinical investigation.
[18] D. Trono,et al. Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis. , 2015, Molecular therapy : the journal of the American Society of Gene Therapy.
[19] O. Ohara,et al. RAG1 Deficiency May Present Clinically as Selective IgA Deficiency , 2015, Journal of Clinical Immunology.
[20] J. McElwee,et al. Identification of Patients with RAG Mutations Previously Diagnosed with Common Variable Immunodeficiency Disorders , 2015, Journal of Clinical Immunology.
[21] L. Notarangelo,et al. A hypomorphic recombination-activating gene 1 (RAG1) mutation resulting in a phenotype resembling common variable immunodeficiency. , 2014, The Journal of allergy and clinical immunology.
[22] M. Seidel. Autoimmune and other cytopenias in primary immunodeficiencies: pathomechanisms, novel differential diagnoses, and treatment. , 2014, Blood.
[23] L. Notarangelo,et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. , 2014, The New England journal of medicine.
[24] L. Notarangelo,et al. Rag Defects and Thymic Stroma: Lessons from Animal Models , 2014, Front. Immunol..
[25] M. van der Burg,et al. Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells , 2014, Nature.
[26] R. Sarwari,et al. Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome. , 2014, The Journal of allergy and clinical immunology.
[27] U. Pannicke,et al. SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. , 2014, Blood.
[28] S. Mantero,et al. Hypomorphic mutation in the RAG2 gene affects dendritic cell distribution and migration , 2013, Journal of leukocyte biology.
[29] E. Haddad,et al. B-cell reconstitution for SCID: should a conditioning regimen be used in SCID treatment? , 2013, The Journal of allergy and clinical immunology.
[30] Aaron R Cooper,et al. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. , 2012, Blood.
[31] G. Wagemaker,et al. Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.
[32] Fabio Grassi,et al. Anti-CD3ε mAb improves thymic architecture and prevents autoimmune manifestations in a mouse model of Omenn syndrome: therapeutic implications. , 2012, Blood.
[33] Y. Takeuchi,et al. Safer, Silencing-Resistant Lentiviral Vectors: Optimization of the Ubiquitous Chromatin-Opening Element through Elimination of Aberrant Splicing , 2012, Journal of Virology.
[34] D. Schatz,et al. V(D)J recombination: mechanisms of initiation. , 2011, Annual review of genetics.
[35] A. Schambach,et al. Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer , 2011, Leukemia.
[36] L. Naldini,et al. Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. , 2011, The Journal of allergy and clinical immunology.
[37] A. Fischer,et al. Immune deficiencies , infection , and systemic immune disorders Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe : Entering a new century , do we do better ? , 2010 .
[38] F. Alt,et al. Expansion of immunoglobulin-secreting cells and defects in B cell tolerance in Rag-dependent immunodeficiency , 2010, The Journal of experimental medicine.
[39] M. van der Burg,et al. Homeostatic expansion of autoreactive immunoglobulin-secreting cells in the Rag2 mouse model of Omenn syndrome , 2010, The Journal of experimental medicine.
[40] R. Buckley. B-cell function in severe combined immunodeficiency after stem cell or gene therapy: a review. , 2010, The Journal of allergy and clinical immunology.
[41] Alessandro Aiuti,et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. , 2009, The New England journal of medicine.
[42] Y. Lokhnygina,et al. Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis. , 2009, The Journal of pediatrics.
[43] M. Cancro. Signalling crosstalk in B cells: managing worth and need , 2009, Nature Reviews Immunology.
[44] L. Notarangelo,et al. Early defects in human T-cell development severely affect distribution and maturation of thymic stromal cells: possible implications for the pathophysiology of Omenn syndrome. , 2009, Blood.
[45] K. Siminovitch,et al. Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.
[46] A. Fischer,et al. Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency. , 2009, Blood.
[47] L. Notarangelo,et al. A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome. , 2007, The Journal of clinical investigation.
[48] N. Olsen,et al. Protein array autoantibody profiles for insights into systemic lupus erythematosus and incomplete lupus syndromes , 2006, Clinical and experimental immunology.
[49] D. Allman,et al. Characterization of marginal zone B cell precursors , 2005, The Journal of experimental medicine.
[50] A. Fischer,et al. AIRE deficiency in thymus of 2 patients with Omenn syndrome. , 2005, The Journal of clinical investigation.
[51] A. Fischer,et al. Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. , 2002, Blood.
[52] J. Puck,et al. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. , 2002, Blood.
[53] K. Rajewsky,et al. Homeostasis of Peripheral B Cells in the Absence of B Cell Influx from the Bone Marrow , 2001, The Journal of experimental medicine.
[54] A. Fischer,et al. Identical mutations in RAG1 or RAG2 genes leading to defective V(D)J recombinase activity can cause either T-B-severe combined immune deficiency or Omenn syndrome. , 2001, Blood.
[55] Sandro Santagata,et al. Partial V(D)J Recombination Activity Leads to Omenn Syndrome , 1998, Cell.
[56] G. Omenn. FAMILIAL RETICULOENDOTHELIOSIS WITH EOSINOPHILIA. , 1965, The New England journal of medicine.
[57] R. Buckley,et al. Post-Transplantation B Cell Function in Different Molecular Types of SCID , 2012, Journal of Clinical Immunology.