John T. Gray

Vertex Pharmaceuticals (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

33h-index7.6kcitations93works0.02yr avg
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Research Topics

Virus-based gene therapy research(54), CRISPR and Genetic Engineering(23), CAR-T cell therapy research(21), Viral Infectious Diseases and Gene Expression in Insects(19), RNA Interference and Gene Delivery(17)

Publications93 total

Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps
Molecular Therapy — Methods & Clinical Development·2022· 24 citedOpen Access
AUTOPHAGIC MYOPATHIES / MYOFIBRILLAR MYOPATHIES / DISTAL MYOPATHIES / POMPE DISEASE
Neuromuscular Disorders·2020
AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy
Molecular Therapy·2019· 25 citedOpen Access
Continued analysis of GAA mice treated with novel hybrid promoter rAAV vectors expressing acid alpha-glucosidase
Molecular Genetics and Metabolism·2019
AAV-mediated gene transfer restores a normal muscle transcriptome in a canine model of X-linked myotubular myopathy
2018· 2 citedOpen Access
METABOLIC MYOPATHIES II
Neuromuscular Disorders·2018
Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome
Molecular Therapy — Methods & Clinical Development·2018· 15 citedOpen Access
TNNT1 nemaline myopathy: natural history and therapeutic frontier
Human Molecular Genetics·2018· 42 citedOpen Access
Establishing the optimal tissue target for alpha-glucosidase gene delivery in Pompe disease
Molecular Genetics and Metabolism·2018
547. AAV Preparations Contain Contamination from DNA Sequences in Production Plasmids Directly Outside of the ITRs
Molecular Therapy·2016· 7 citedOpen Access
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
Science Translational Medicine·2016· 284 citedOpen Access
Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency
Blood·2015· 6 cited
High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease
Blood·2015· 3 cited
Induction of Neuron-Specific Degradation of Coenzyme A Models Pantothenate Kinase-Associated Neurodegeneration by Reducing Motor Coordination in Mice
PLoS ONE·2015· 27 citedOpen Access
81. Development of a Second Generation Stable Lentiviral Packaging Cell Line in Support of Clinical Gene Transfer Protocols
Molecular Therapy·2015· 9 citedOpen Access
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
New England Journal of Medicine·2014· 1.2k citedOpen Access
Manufacturing viral gene therapy vectors: general approaches and challenges
˜The œbiomedical & life sciences collection.·2014· 1 cited
Chimeric antigen receptor-redirected CD45RA-negative T cells have potent antileukemia and pathogen memory response without graft-versus-host activity
Leukemia·2014· 56 citedOpen Access
False-Positive HIV PCR Test Following Ex Vivo Lentiviral Gene Transfer Treatment of X-linked Severe Combined Immunodeficiency Vector
Molecular Therapy·2014· 17 citedOpen Access
Non-Culture-Based Confirmation of Complementary Methods for Immunoagglutination and PCR as Ultrasound-Enhanced Latex
2014
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Frequent Co-authors

Andrew M. Davidoff(18), Amit C. Nathwani(15), Catherine Y. Ng(13), Arthur W. Nienhuis(13), Jenny McIntosh(9), Edward G. D. Tuddenham(8), Junfang Zhou(8), Yunyu Spence(8), Christopher L. Morton(7), Brian P. Sorrentino(7), Derek A. Persons(7), Cecilia Rosales(6), James A. Allay(6), Susan Sleep(6), Katherine A. High(6), Federico Mingozzi(5), Robert E. Throm(5), Sheng Zhou(5), Anne Riddell(4), Mark A. Kay(4)