Gene therapy clinical trials worldwide to 2012 - an update

602. Dose-Dependent Activation of Capsid-Specific T Cells after AAV Serotype 8 Vector Administration in a Clinical Study for Hemophilia B

(2016)   MOLECULAR THERAPY

Extended Survival of Glioblastoma Patients After Chemoprotective HSC Gene Therapy

(2012) J. E. Adair et al.   Science Translational Medicine

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Tumor Regression in Patients With Metastatic Synovial Cell Sarcoma and Melanoma Using Genetically Engineered Lymphocytes Reactive With NY-ESO-1

(2011) Paul F. Robbins et al.   JOURNAL OF CLINICAL ONCOLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial

(2011) Hyoung Jin Kang et al.   MOLECULAR THERAPY

Solving the Problem of γ-Retroviral Vectors Containing Long Terminal Repeats

(2011) Derek A Persons et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia

(2011) David L. Porter et al.   NEW ENGLAND JOURNAL OF MEDICINE

T Cells with Chimeric Antigen Receptors Have Potent Antitumor Effects and Can Establish Memory in Patients with Advanced Leukemia

(2011) M. Kalos et al.   Science Translational Medicine

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

Update on gene therapy for immunodeficiencies

(2010) Donald B. Kohn   CLINICAL IMMUNOLOGY

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency

(2010) Francesca Ferrua et al.   Current Opinion in Allergy and Clinical Immunology

Bone Marrow Transplantation and Alternatives for Adenosine Deaminase Deficiency

(2010) H. Bobby Gaspar   IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA

Gene Therapy for SCID-X1: Round 2

(2010) Roland W Herzog   MOLECULAR THERAPY

Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma

(2010) Manuel Grez et al.   MOLECULAR THERAPY

Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells

(2010) Giorgia Santilli et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations

(2010) Sarah Forrest et al.   NEUROMUSCULAR DISORDERS

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

Restoration of NET formation by gene therapy in CGD controls aspergillosis

(2009) M. Bianchi et al.   BLOOD

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

(2009) E. M. Kang et al.   BLOOD

How I treat ADA deficiency

(2009) H. B. Gaspar et al.   BLOOD

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

(2009) A. Annoni et al.   BLOOD

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

(2009) Dohoon Kim et al.   Cell Stem Cell

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy

(2009) Yasuhiro Kazuki et al.   MOLECULAR THERAPY

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya et al.   NATURE

Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

(2009) Artur V. Cideciyan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome

(2009) Noemi FUSAKI et al.   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Human Induced Pluripotent Stem Cells Free of Vector and Transgene Sequences

(2009) J. Yu et al.   SCIENCE

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Adenoviral Gene Delivery Can Reprogram Human Fibroblasts to Induced Pluripotent Stem Cells

(2009) Wenbo Zhou et al.   STEM CELLS

Recent advances in gene therapy for severe congenital immunodeficiency diseases

(2008) Robert Sokolic et al.   CURRENT OPINION IN HEMATOLOGY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease

(2008) M. Wernig et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA