Perspectives on Best Practices for Gene Therapy Programs

被引：13

作者：

Cheever, Thomas R. ^{[1
]}

Berkley, Dale ^{[2
]}

Braun, Serge ^{[3
]}

Brown, Robert H. ^{[4
]}

Byrne, Barry J. ^{[5
]}

Chamberlain, Jeffrey S. ^{[6
]}

Cwik, Valerie ^{[7
]}

Duan, Dongsheng ^{[8
]}

Federoff, Howard J. ^{[9
]}

High, Katherine A. ^{[10
,11
,12
]}

Kaspar, Brian K. ^{[13
]}

Klinger, Katherine W. ^{[14
]}

Larkindale, Jane ^{[15
]}

Lincecum, John ^{[16
]}

Mavilio, Fulvio ^{[17
]}

McDonald, Cheryl L. ^{[18
]}

McLaughlin, James ^{[19
]}

McLeod, Bonnie Weiss ^{[20
]}

Mendell, Jerry R. ^{[13
]}

Nuckolls, Glen ^{[1
]}

Stedman, Hansell H. ^{[21
]}

Tagle, Danilo A. ^{[22
]}

Vandenberghe, Luk H. ^{[23
,24
]}

Wang, Hao ^{[1
]}

Wernett, Pamela J. ^{[1
]}

Wilson, James M. ^{[25
]}

Porter, John D. ^{[1
]}

Gubitz, Amelie K. ^{[1
]}

机构：

[1] NINDS, NIH, Bethesda, MD 20892 USA

[2] NIH, Off Gen Counsel, Bethesda, MD 20892 USA

[3] Assoc Francaise Myopathies, F-91002 Evry, France

[4] Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01655 USA

[5] Univ Florida, Sch Med, Dept Pediat, Gainesville, FL 32610 USA

[6] Univ Washington, Dept Neurol, Seattle, WA 98195 USA

[7] Muscular Dystrophy Assoc, Tucson, AZ 85718 USA

[8] Univ Missouri, Dept Mol Microbiol & Immunol, Columbia, MO 65212 USA

[9] Georgetown Univ, Med Ctr, Dept Neurol, Washington, DC 20057 USA

[10] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

[11] Univ Penn, Howard Hughes Med Inst, Philadelphia, PA 19104 USA

[12] Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA

[13] Nationwide Childrens Hosp, Res Inst, Ctr Gene Therapy, Columbus, OH 43205 USA

[14] Genzyme Corp, Framingham, MA 01701 USA

[15] NMD Consulting, Tucson, AZ 85750 USA

[16] Amyotroph Lateral Sclerosis Therapy Dev Inst, Cambridge, MA 02139 USA

[17] Genethon, F-91002 Evry, France

[18] NHLBI, NIH, Bethesda, MD 20892 USA

[19] Voyager Therapeut, Cambridge, MA 02139 USA

[20] Cooley LLP, Washington, DC 20001 USA

[21] Univ Penn, Perelman Sch Med, Dept Surg, Philadelphia, PA 19104 USA

[22] NIH, Natl Ctr Adv Translat Sci, Bethesda, MD 20892 USA

[23] Massachusetts Eye & Ear Infirm, Boston, MA 02114 USA

[24] Harvard Univ, Sch Med, Dept Ophthalmol, Boston, MA 02114 USA

[25] Univ Penn, Gene Therapy Program, Philadelphia, PA 19104 USA

来源：

HUMAN GENE THERAPY | 2015年 / 26卷 / 03期

关键词：

D O I：

10.1089/hum.2014.147

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

With recent successes in gene therapy trials for hemophilia and retinal diseases, the promise and prospects for gene therapy are once again garnering significant attention. To build on this momentum, the National Institute of Neurological Disorders and Stroke and the Muscular Dystrophy Association jointly hosted a workshop in April 2014 on "Best Practices for Gene Therapy Programs," with a focus on neuromuscular disorders. Workshop participants included researchers from academia and industry as well as representatives from the regulatory, legal, and patient advocacy sectors to cover the gamut from preclinical optimization to intellectual property concerns and regulatory approval. The workshop focused on three key issues in the field: (1) establishing adequate scientific premise for clinical trials in gene therapy, (2) addressing regulatory process issues, and (3) intellectual property and commercialization issues as they relate to gene therapy. The outcomes from the discussions at this workshop are intended to provide guidance for researchers and funders in the gene therapy field.

引用

页码：127 / 133

页数：7

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