title: Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.
abstract: Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors. The Gene Therapy for Rare Diseases workshop, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities and Office of Rare Diseases Research, brought together investigators from different disciplines to discuss the challenges and opportunities for advancing the field including means for enhancing data sharing for preclinical and clinical studies, development and utilization of available NIH resources, and interactions with the U.S. Food and Drug Administration.
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD 20892, USA.
authors.forename: Marina
authors.initials: M
authors.lastname: O'Reilly
authors.forename: Donald B
authors.initials: DB
authors.lastname: Kohn
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Bartlett
authors.forename: Janet
authors.initials: J
authors.lastname: Benson
authors.forename: Philip J
authors.initials: PJ
authors.lastname: Brooks
authors.forename: Barry J
authors.initials: BJ
authors.lastname: Byrne
authors.forename: Carlos
authors.initials: C
authors.lastname: Camozzi
authors.forename: Kenneth
authors.initials: K
authors.lastname: Cornetta
authors.forename: Ronald G
authors.initials: RG
authors.lastname: Crystal
authors.forename: Yuman
authors.initials: Y
authors.lastname: Fong
authors.forename: Linda
authors.initials: L
authors.lastname: Gargiulo
authors.forename: Rashmi
authors.initials: R
authors.lastname: Gopal-Srivastava
authors.forename: Katherine A
authors.initials: KA
authors.lastname: High
authors.forename: Samuel G
authors.initials: SG
authors.lastname: Jacobson
authors.forename: Robert C
authors.initials: RC
authors.lastname: Jambou
authors.forename: Maureen
authors.initials: M
authors.lastname: Montgomery
authors.forename: Eugene
authors.initials: E
authors.lastname: Rosenthal
authors.forename: R Jude
authors.initials: RJ
authors.lastname: Samulski
authors.forename: Sonia I
authors.initials: SI
authors.lastname: Skarlatos
authors.forename: Brian
authors.initials: B
authors.lastname: Sorrentino
authors.forename: James M
authors.initials: JM
authors.lastname: Wilson
authors.forename: Yun
authors.initials: Y
authors.lastname: Xie
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Corrigan-Curay
mesh_terms: D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D006801:Humans; D009316:National Institutes of Health (U.S.); D035583:Rare Diseases / Q000235:genetics* / Q000628:therapy*; D057170:Translational Research, Biomedical; D014481:United States; D014486:United States Food and Drug Administration