title: The National Institutes of Health Oversight of Human Gene Transfer Research: Enhancing Science and Safety.
abstract: The National Institutes of Health (NIH) oversight of human gene transfer research, which is defined as the deliberate transfer of recombinant and/or synthetic nucleic acid molecules to humans, originates with the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines). The NIH Guidelines, which were first published in the Federal Register almost 40 years ago, have been amended numerous times to remain responsive to scientific progress and to clearly define the responsibilities of NIH, the Recombinant DNA Advisory Committee (RAC), investigators, and institutions. Human gene transfer trials conducted at clinical sites in the United States (USA) are subject to the NIH Guidelines if they are conducted at, or sponsored by, an institution that receives any support for recombinant or synthetic nucleic acid research from the NIH. Human gene transfer trials conducted either in the USA or abroad are also subject to the NIH Guidelines if the investigational agent was developed with NIH funds and the institution that developed the investigational materials sponsors or participates in these projects. Trials are registered with the NIH Office Biotechnology Activities (OBA) and there are ongoing reporting requirements. Each new trial is reviewed by the RAC, and those that are novel or raise unique ethical or social issues are selected for review at quarterly public RAC meetings. The RAC also advises the NIH on policy and other matters relating to clinical gene transfer research and biosafety.
authors.affiliation: Program on Biosecurity and Biosafety Policy, Office of Science Policy, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Marina
authors.initials: M
authors.lastname: O'Reilly
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Robert
authors.initials: R
authors.lastname: Jambou
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Eugene
authors.initials: E
authors.lastname: Rosenthal
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Maureen
authors.initials: M
authors.lastname: Montgomery
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Morad
authors.initials: M
authors.lastname: Hassani
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Linda
authors.initials: L
authors.lastname: Gargiulo
authors.affiliation: Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD, USA. corrigaja@nhlbi.nih.gov.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Corrigan-Curay
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D004274:DNA, Recombinant / Q000627:therapeutic use*; D004335:Drug and Narcotic Control / Q000331:legislation & jurisprudence*; D015316:Genetic Therapy / Q000941:ethics / Q000331:legislation & jurisprudence*; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D007258:Informed Consent / Q000941:ethics / Q000331:legislation & jurisprudence; D009316:National Institutes of Health (U.S.); D061214:Patient Safety / Q000331:legislation & jurisprudence; D017410:Practice Guidelines as Topic; D012107:Research Design; D057170:Translational Research, Biomedical / Q000941:ethics / Q000331:legislation & jurisprudence*; D014481:United States
chemical_list: D004274:DNA, Recombinant
keywords: Adverse event database; Clinical trials; Delivery system; GeMCRIS; Human gene transfer; Informed consent; Institute of Medicine; NIH Guidelines; National Institutes of Health; Office of Biotechnology Activities; Office of Science Policy; Recombinant DNA Advisory Committee; Safety data; Scientific review