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.
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Notes
- 1.
Trials conducted abroad are 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.
- 2.
OHRP is a part of the US Department of Health and Human Services (HHS) and provides leadership in the protection of the rights, welfare, and well-being of subjects involved in research conducted or supported by HHS. Additional information regarding OHRP may be found at this URL: http://www.hhs.gov/ohrp/index.html
- 3.
Although establishment of an IBC for review of gene transfer and other research is a requirement of NIH funding, other US government agencies that do not receive NIH funding, such as the Department of Veterans Affairs and Department of Defense, certain private institutions, and companies (which also do not receive NIH funding) require that their research be conducted in accordance with the NIH Guidelines, including the establishment of an IBC for review and approval of human gene transfer trials.
Abbreviations
- AAV:
-
Adeno-associated virus
- ASGCT:
-
American Society of Gene and Cell Therapy
- FDA:
-
Food and Drug Administration
- GeMCRIS:
-
Genetic Modification Clinical Research Information System
- GTSAB:
-
Gene Transfer Safety Assessment Board
- HSV:
-
Herpes simplex virus
- IBC:
-
Institutional Biosafety Committee
- IND:
-
Investigational New Drug
- IOM:
-
Institute of Medicine
- IRB:
-
Institutional Review Board
- NIH:
-
National Institutes of Health
- OBA:
-
Office of Biotechnology Activities
- OHRP:
-
Office of Human Research Protection
- PI:
-
Principal Investigator
- RAC:
-
Recombinant DNA Advisory Committee
- SAE:
-
Serious adverse event
- SB:
-
Sleeping Beauty
- VSV:
-
Vesicular stomatitis virus
- X-SCID:
-
X-linked severe combined immunodeficiency
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O’Reilly, M. et al. (2015). The National Institutes of Health Oversight of Human Gene Transfer Research: Enhancing Science and Safety. In: Galli, M., Serabian, M. (eds) Regulatory Aspects of Gene Therapy and Cell Therapy Products. Advances in Experimental Medicine and Biology(), vol 871. Springer, Cham. https://doi.org/10.1007/978-3-319-18618-4_2
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DOI: https://doi.org/10.1007/978-3-319-18618-4_2
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