C. Walsh

发表

New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies.

C. Walsh, 2004, Seminars in hematology.

Congenital bleeding disorders.

N. Key, M. Rick, C. Walsh, 2003, Hematology. American Society of Hematology. Education Program.

Hemophilia Gene Therapy

C. Walsh, 2015 .

Targeted integration of a rAAV vector into the AAVS1 region.

P. Ward, C. Walsh, 2012, Virology.

Sustained expression of human factor VIII in mice using a parvovirus-based vector.

A. Bruce, C. Walsh, H. Chao, 2000, Blood.

Induction of tolerance to human factor VIII in mice.

C. Walsh, H. Chao, Christopher E. Walsh, 2001, Blood.

Persistent expression of canine factor IX in hemophilia B canines

R. Samulski, D. Bellinger, T. Nichols, 1999, Gene Therapy.

Fanconi anemia and novel strategies for therapy.

M. Buchwald, J. Liu, C. Walsh, 1994, Blood.

A functionally active retrovirus vector for gene therapy in Fanconi anemia group C

M. Buchwald, N. Young, A. Nienhuis, 1994 .

Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC).

N. Young, E. Read, S. Leitman, 1999, Human gene therapy.

Adeno-associated Virus Serotype 8 ApoA-I Gene Transfer Reduces Progression of Atherosclerosis in ApoE-KO Mice: Comparison of Intramuscular and Intravenous Administration

V. Fuster, J. Badimón, C. Giannarelli, 2011, Journal of cardiovascular pharmacology.

Recombinant long‐acting glycoPEGylated factor IX (nonacog beta pegol) in haemophilia B: assessment of target joints in multinational phase 3 clinical trials

C. Négrier, G. Young, P. Collins, 2016, Haemophilia : the official journal of the World Federation of Hemophilia.

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

M. Garcia-Blanco, L. Mitchell, S. Mansfield, 2003, Nature Medicine.

Ledipasvir–sofosbuvir and sofosbuvir plus ribavirin in patients with chronic hepatitis C and bleeding disorders

P. Sax, K. Reddy, K. Workowski, 2017, Haemophilia : the official journal of the World Federation of Hemophilia.

Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector.

J. Olsen, C. Walsh, K. Yamada, 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

Phenotypes of Allo- and Autoimmune Antibody Responses to FVIII Characterized by Surface Plasmon Resonance

M. Epstein, K. Lewis, C. Kempton, 2013, PloS one.

A Phase II Open-Label Study Evaluating Hemostatic Activity, Pharmacokinetics and Safety of Recombinant Porcine Factor VIII (OBI-1) in Hemophilia A Patients with Alloantibody Inhibitors Directed Against Human FVIII.

C. Kempton, C. Kessler, B. Ritchie, 2007 .

Identified unmet needs and proposed solutions in mild‐to‐moderate haemophilia: A summary of opinions from a roundtable of haemophilia experts

D. Cooper, M. Witkop, L. Boggio, 2021, Haemophilia : the official journal of the World Federation of Hemophilia.

Acquired hemophilia: a rare but life-threatening potential cause of bleeding in the intensive care unit

A. Shander, C. Cromwell, C. Walsh, 2011, Intensive Care Medicine.

Gene therapy for hemophilia: are viral vectors really feasible?

C. Walsh, 2003, Journal of thrombosis and haemostasis : JTH.

Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector.

R. Samulski, N. Young, A. Nienhuis, 1994, The Journal of clinical investigation.

Acquired hemophilia presenting as profound hematuria: evaluation, diagnosis, and management of elusive cause of bleeding in the emergency department setting.

A. Shander, C. Cromwell, H. Bailey, 2013, The Journal of emergency medicine.

A trial of recombinant human interleukin‐1 in patients with severe refractory aplastic anaemia

N. Young, A. Nienhuis, S. Anderson, 1992, British journal of haematology.

Retroviral gene transfer for the assignment of Fanconi anemia (FA) patients to a FA complementation group

Johnson M Liu, M. Digweed, C. Walsh, 1998, Human Genetics.

Functional correction of Fanconi anemia group A hematopoietic cells by retroviral gene transfer.

H. Joenje, J. Liu, C. Walsh, 1997, Blood.

A functionally active retrovirus vector for gene therapy in Fanconi anemia group C.

M. Buchwald, N. Young, A. Nienhuis, 1994, Blood.

Prophylaxis for adults with severe haemophilia: a compelling need for evidence‐based guidelines

C. Walsh, 2008, Haemophilia : the official journal of the World Federation of Hemophilia.

Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practices

L. Valentino, C. Walsh, C. E. Walsh, 2009, Haemophilia : the official journal of the World Federation of Hemophilia.

Perioperative management of haemophilia B: A critical appraisal of the evidence and current practices

E. Neufeld, P. Giangrande, D. Cooper, 2017, Haemophilia : the official journal of the World Federation of Hemophilia.

Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial.

J. Oldenburg, C. Négrier, J. Mahlangu, 2014, Blood.

Intrathecal gene transfer by adeno-associated virus for pain.

M. Banck, A. Beutler, E. Milligan, 2005, Current opinion in molecular therapeutics.

Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats

J. Morrison, W. Janssen, M. Banck, 2006, Molecular pain.

Management of US men, women, and children with hemophilia and methods and demographics of the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B‐HERO‐S) study

David B Clark, T. Buckner, D. Cooper, 2017, European journal of haematology.

Current and future prospects for hemophilia gene therapy

P. Ward, C. Walsh, 2016, Expert review of hematology.

Impact of inhibitors on hemophilia a mortality in the United States

C. Miller, J. Soucie, C. Walsh, 2015, American journal of hematology.

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors.

R. Samulski, P. Monahan, Y. Liu, 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

The burden of inhibitors in haemophilia patients

V. Jiménez‐Yuste, S. Grancha, G. Auerswald, 2016, Thrombosis and Haemostasis.

Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.

R. Montgomery, S. Fahs, Q. Shi, 2010, Blood.

Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) for prophylaxis and treatment of bleeding episodes in patients with haemophilia B

J. Oldenburg, C. Négrier, J. Mahlangu, 2013 .

New high‐technology products for the treatment of haemophilia

S. Pipe, J. Saint-Remy, C. Walsh, 2004, Haemophilia : the official journal of the World Federation of Hemophilia.

greater in adults ≥ 30 years . 9 Treatment Strategies Immune Tolerance Induction For inhibitor patients , the best treatment option is eradication of the inhibitor through immune tolerance

Leonard, M. Recht, P. Mathew, 2022 .

Emicizumab for the Treatment of Acquired Hemophilia a: A Multicenter US Case Series

C. Kessler, R. Kruse-Jarres, L. Malec, 2021, Blood.

Efficacy of EHL N9-GP for on-demand treatment of bleeding episodes in hemophilia B: analysis of pivotal trial data

D. Cooper, G. Young, M. Escobar, 2019, Journal of blood medicine.

Optimal trough levels in haemophilia B: Raising expectations

M. Escobar, C. Walsh, M. Coppens, 2020, Haemophilia : the official journal of the World Federation of Hemophilia.

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression.

R. Samulski, B. Konkle, I. Bilic, 2020, Blood.

Expression of human factor VIII by splicing between dimerized AAV vectors.

Xiao Xiao, A. Bruce, C. Walsh, 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

Hemophilia clinical gene therapy: brief review.

C. Walsh, Katherine M Batt, 2013, Translational research : the journal of laboratory and clinical medicine.

Recombinant B‐domain‐deleted porcine sequence factor VIII (r‐pFVIII) for the treatment of bleeding in patients with congenital haemophilia A and inhibitors

J. Mahlangu, N. Key, C. Walsh, 2017, Haemophilia : the official journal of the World Federation of Hemophilia.

Eradication of factor viii inhibitors in patients with mild and moderate hemophilia A

C. Kempton, J. Soucie, G. Young, 2012, American journal of hematology.

Patient‐ and caregiver‐reported bleeding symptoms and reasons for starting and stopping treatment with recombinant factor VIIa: analysis of the Dosing Observational Study in Haemophilia (DOSE)

L. Valentino, D. Cooper, G. Young, 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.

R. Kole, S. Fucharoen, H. Sierakowska, 2002, Molecular pharmacology.

Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

R. Samulski, N. Young, A. Nienhuis, 1992, Proceedings of the National Academy of Sciences of the United States of America.

Fanconi anemia C protein acts at a switch between apoptosis and necrosis in mitomycin C-induced cell death.

C. Guillouf, F. Rosselli, E. Moustacchi, 1999, Experimental cell research.

Retroviral vector for gene therapy of X-linked severe combined immunodeficiency syndrome.

W. Leonard, M. Noguchi, M. Qazilbash, 1995, Journal of hematotherapy.

Retroviral-mediated transduction of the fanconi anemia C complementing (FACC) gene in two murine transplantation models.

J. Liu, S. Kim, C. Walsh, 1995, Blood cells, molecules & diseases.

Retroviral mediated gene transfer of the Fanconi anemia complementation group C gene to hematopoietic progenitors of group C patients.

N. Young, A. Barrett, C. Dunbar, 1997, Human gene therapy.

Human α Spectrin II and the Fanconi Anemia Proteins FANCA and FANCC Interact to Form a Nuclear Complex*

M. W. Lambert, L. McMahon, C. Walsh, 1999, The Journal of Biological Chemistry.

A deficiency in a 230 kDa DNA repair protein in fanconi anemia complementation group A cells is corrected by the FANCA cDNA.

M. W. Lambert, L. McMahon, C. Walsh, 1999, Carcinogenesis.

Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer.

V. Madden, D. Mccarty, C. Walsh, 2003, BioTechniques.

Intracellular localization of the Fanconi anemia complementation group A protein.

D. Simpson, M. Yountz, C. Walsh, 1999, Biochemical and biophysical research communications.

Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector.

W. Ebell, R. Hawley, F. Arwert, 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene Therapy for Human Hemoglobinopathies

Jeffery L. Miller, R. Samulski, A. Nienhuis, 1993, Proceedings of the Society for Experimental Biology and Medicine. Society for Experimental Biology and Medicine.

Gene therapy for the hemophilias.

C. Walsh, 2002, Current opinion in pediatrics.

Endogenous Canine FIX Antigen Exists in Chapel Hill Strain Hemophilia B Canine

C. Walsh, H. Chao, 1999, Thrombosis and Haemostasis.

Gene therapy Progress and Prospects: Gene therapy for the hemophilias

Christopher E Walsh, C. Walsh, 2003, Gene Therapy.

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors.

R. Samulski, Chengwen Li, J. Rabinowitz, 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

Single-copy transduction and expression of human gamma-globin in K562 erythroleukemia cells using recombinant adeno-associated virus vectors: the effect of mutations in NF-E2 and GATA-1 binding motifs within the hypersensitivity site 2 enhancer.

R. Samulski, A. Nienhuis, P. Ney, 1993, Blood.

RNA repair using spliceosome-mediated RNA trans-splicing.

S. Mansfield, C. Walsh, H. Chao, 2004, Trends in molecular medicine.

Phenotypic correction of Fanconi anemia group C knockout mice.

C. Walsh, K. Gush, M. Grompe, 2000, Blood.

Safe and Sustained Overexpression of Functional Apolipoprotein A-I/High-density Lipoprotein in Apolipoprotein A-I–null Mice by Muscular Adeno-associated Viral Serotype 8 Vector Gene Transfer

V. Fuster, J. Badimón, C. Giannarelli, 2009, Journal of cardiovascular pharmacology.

RNA repair for haemophilia A

Hengjun Chao, Christopher E Walsh, C. Walsh, 2006, Expert Reviews in Molecular Medicine.

Absence of Inhibitor Development in the Routine Clinical Use of Alphanate® in Patients with Severe Hemophilia A: Results From a Long-Term Post-Marketing Surveillance Study.

K. Saxena, M. Escobar, A. Páez, 2009 .

Spliceosome-mediated RNA trans-splicing.

Yanping Yang, C. Walsh, 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

The Fanconi anemia complementation group C gene (FAC) suppresses transformation of mutant fibroblasts by the SV40 virus.

S. Kajigaya, J. Liu, C. Walsh, 1996, Biochemical and biophysical research communications.

AAV vectors for hemophilia B gene therapy.

C. Walsh, H. Chao, 2004, The Mount Sinai journal of medicine, New York.

Title Endogenous factor VIII synthesis from the intron 22-inverted F 8 locus may modulate the immunogenicity of replacement therapy for hemophilia

Yasmin, C. Yanover, T. Przytycka, 2014 .

Chimeric AAV Cap sequences alter gene transduction.

P. Ward, C. Walsh, 2009, Virology.

Adeno-associated Virus Serotype 8 ApoA-I Gene Transfer Reduces Progression of Atherosclerosis in ApoE-KO Mice: Comparison of Intramuscular and Intravenous Administration

V. Fuster, J. Badimón, C. Giannarelli, 2011, Journal of cardiovascular pharmacology.