title: Perspectives on best practices for gene therapy programs.
abstract: 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.
authors: 1 National Institute of Neurological Disorders and Stroke, National Institutes of Health , Bethesda, MD 20892.
authors: Thomas R
authors: TR
authors: Cheever
authors: Dale
authors: D
authors: Berkley
authors: Serge
authors: S
authors: Braun
authors: Robert H
authors: RH
authors: Brown
authors: Barry J
authors: BJ
authors: Byrne
authors: Jeffrey S
authors: JS
authors: Chamberlain
authors: Valerie
authors: V
authors: Cwik
authors: Dongsheng
authors: D
authors: Duan
authors: Howard J
authors: HJ
authors: Federoff
authors: Katherine A
authors: KA
authors: High
authors: Brian K
authors: BK
authors: Kaspar
authors: Katherine W
authors: KW
authors: Klinger
authors: Jane
authors: J
authors: Larkindale
authors: John
authors: J
authors: Lincecum
authors: Fulvio
authors: F
authors: Mavilio
authors: Cheryl L
authors: CL
authors: McDonald
authors: James
authors: J
authors: McLaughlin
authors: Bonnie
authors: B
authors: Weiss McLeod
authors: Jerry R
authors: JR
authors: Mendell
authors: Glen
authors: G
authors: Nuckolls
authors: Hansell H
authors: HH
authors: Stedman
authors: Danilo A
authors: DA
authors: Tagle
authors: Luk H
authors: LH
authors: Vandenberghe
authors: Hao
authors: H
authors: Wang
authors: Pamela J
authors: PJ
authors: Wernett
authors: James M
authors: JM
authors: Wilson
authors: John D
authors: JD
authors: Porter
authors: Amelie K
authors: AK
authors: Gubitz
mesh_terms: D002986:Clinical Trials as Topic; D015316:Genetic Therapy / Q000331:legislation & jurisprudence / Q000379:methods* / Q000592:standards*; D033161:Government Regulation; D006801:Humans; D018973:Intellectual Property; D009468:Neuromuscular Diseases / Q000235:genetics* / Q000628:therapy*
references: JAMA. 2005 Aug 17;294(7):826-32
references: 16106008
references: Sci Am. 2014 Mar;310(3):52-7
references: 24660328
references: Hum Gene Ther Clin Dev. 2013 Jun;24(2):55-64
references: 23808604
references: Nature. 2012 Oct 11;490(7419):187-91
references: 23060188