Perspectives on Best Practices for Gene Therapy Programs

Human Gene Therapy

Volumn 26, Issue 3, 2015, Pages 127-133

  Cheever, Thomas R ^a   Berkley, Dale ^b   Braun, Serge ^c   Brown, Robert H ^d   Byrne, Barry J ^e   Chamberlain, Jeffrey S ^f   Cwik, Valerie ^g   Duan, Dongsheng ^h   Federoff, Howard J ⁱ   High, Katherine A ^j,k   Kaspar, Brian K ^l   Klinger, Katherine W ^m   Larkindale, Jane ⁿ   Lincecum, John ^o   Mavilio, Fulvio ^p   McDonald, Cheryl L ^q   McLaughlin, James ^r   Weiss McLeod, Bonnie ^s   Mendell, Jerry R ^l   Nuckolls, Glen ^a   Stedman, Hansell H ^k   Tagle, Danilo A ^t   Vandenberghe, Luk H ^u,v   Wang, Hao ^a   Wernett, Pamela J ^a   Wilson, James M ^w   Porter, John D ^a   Gubitz, Amelie K ^a   more..

^a NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c Association Francaise Contre les Myopathies   (France)

^d UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^e UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^f UNIVERSITY OF WASHINGTON   (United States)

^g Muscular Dystrophy Association   (United States)

^h University of Missouri   (United States)

ⁱ GEORGETOWN UNIVERSITY MEDICAL CENTER   (United States)

^j HOWARD HUGHES MEDICAL INSTITUTE   (United States)

^k UNIVERSITY OF PENNSYLVANIA   (United States)

^l THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^m SANOFI   (France)

ⁿ NMD Consulting   (United States)

^o Amyotrophic Lateral Sclerosis Therapy Development Institute   (United States)

^p GÉNÉTHON   (France)

^q NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^r VOYAGER THERAPEUTICS   (United States)

^s Harvard Medical School ^*  (United States)

^t NATIONAL INSTITUTES OF HEALTH   (United States)

^u MASSACHUSETTS EYE AND EAR INFIRMARY   (United States)

^v HARVARD MEDICAL SCHOOL   (United States)

^w UNIVERSITY OF PENNSYLVANIA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CLINICAL RESEARCH; CLINICAL TRIAL (TOPIC); GENE DELIVERY SYSTEM; GENE THERAPY; HEALTH PROGRAM; HEMOPHILIA; HUMAN; MEDICAL PRACTICE; NEUROMUSCULAR DISEASE; NONHUMAN; PATENT; RETINA DISEASE; GENETICS; GOVERNMENT REGULATION; LEGISLATION AND JURISPRUDENCE; NEUROMUSCULAR DISEASES; PROCEDURES; STANDARDS;

CLINICAL TRIALS AS TOPIC; GENETIC THERAPY; GOVERNMENT REGULATION; HUMANS; INTELLECTUAL PROPERTY; NEUROMUSCULAR DISEASES;

EID: 84924937245     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.147     Document Type: Article

References (4)

1. Lewis R. Gene therapy's second act. Sci Am 2014;310:52-57.
2. Landis SC, Amara SG, Asadullah K, et al. A call for transparent reporting to optimize the predictive value of preclinical research. Nature 2012;490:187-191.
3. Bryant LM, Christopher DM, Giles AR, et al. Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev 2013;24:55-64.
4. Wendler D, Belsky L, Thompson KM, et al. Quantifying the federal minimal risk standard: implications for pediatric research without a prospect of direct benefit. JAMA 2015;294:826-832.