Published in Mol Ther Methods Clin Dev on May 25, 2016
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | NCT00643747
CAR T Cell Receptor Immunotherapy for Patients With B-cell Lymphoma | NCT00924326
Gene Therapy for Blindness Caused by Choroideremia | NCT01461213
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | NCT02122952
Gene Therapy for Metachromatic Leukodystrophy (TIGET-MLD) | NCT01560182
A Study of AAV-hAADC-2 in Subjects With Parkinson's Disease | NCT00229736
Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS) | NCT01515462
Redirected Auto T Cells for Advanced Myeloma | NCT01352286
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) | NCT01175239
Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency | NCT01380990
Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL) | NCT02453477
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID2) | NCT01410019
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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature (2013) 1.80
Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease. Hum Gene Ther (2012) 1.70
A comprehensive review of retinal gene therapy. Mol Ther (2013) 1.70
A clinically adaptable method to enhance the cytotoxicity of natural killer cells against B-cell malignancies. Cytotherapy (2012) 1.67
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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature (2014) 1.57
Overcoming preexisting humoral immunity to AAV using capsid decoys. Sci Transl Med (2013) 1.56
Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1. Blood (2015) 1.52
Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates. Hum Gene Ther (2013) 1.52
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. Blood (2012) 1.48
Oncolytic viruses: a new class of immunotherapy drugs. Nat Rev Drug Discov (2015) 1.47
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Blood (2012) 1.46
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med (2014) 1.42
Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther (2010) 1.34
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