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.
Subject(s)
DNA, Recombinant/therapeutic use , Drug and Narcotic Control/legislation & jurisprudence , Genetic Therapy/legislation & jurisprudence , Genetic Vectors/therapeutic use , Translational Research, Biomedical/legislation & jurisprudence , Animals , Clinical Trials as Topic , Genetic Therapy/ethics , Humans , Informed Consent/ethics , Informed Consent/legislation & jurisprudence , National Institutes of Health (U.S.) , Patient Safety/legislation & jurisprudence , Practice Guidelines as Topic , Research Design , Translational Research, Biomedical/ethics , United StatesABSTRACT
Recently, the gene therapy field has begun to experience clinical successes in a number of different diseases using various approaches and vectors. The workshop Gene Therapy: Charting a Future Course, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities, brought together early and mid-career researchers to discuss the key scientific challenges and opportunities, ethical and communication issues, and NIH and foundation resources available to facilitate further clinical advances.
Subject(s)
Genetic Therapy/ethics , Animals , Education, Continuing , Genetic Research , Genetic Therapy/economics , Genetic Therapy/legislation & jurisprudence , Genetic Vectors , Humans , National Institutes of Health (U.S.) , Stem Cell Research , Stem Cell Transplantation , Transduction, Genetic , United StatesABSTRACT
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.
Subject(s)
Rare Diseases/genetics , Rare Diseases/therapy , Clinical Trials as Topic , Genetic Therapy , Humans , National Institutes of Health (U.S.) , Translational Research, Biomedical , United States , United States Food and Drug AdministrationSubject(s)
Genetic Therapy , Genetic Vectors , Hematopoietic Stem Cell Transplantation , Retroviridae/genetics , Gene Transfer Techniques , Genetic Therapy/adverse effects , Genetic Therapy/ethics , Genetic Therapy/legislation & jurisprudence , Genetic Vectors/adverse effects , Genetic Vectors/genetics , Humans , Research DesignABSTRACT
In response to public and scientific concerns regarding human gene transfer research, the National Institutes of Health (NIH) developed a transparent oversight system that extends to human gene transfer protocols that are either conducted with NIH funding or conducted at institutions that receive NIH funding for recombinant DNA research. The NIH Recombinant DNA Advisory Committee (RAC) has been the primary advisory body to NIH regarding the conduct of this research. Human gene transfer research proposals that are subject to the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines) must be submitted to the NIH Office of Biotechnology Activities (OBA), and protocols that raise novel scientific, safety, medical, ethical, or social issues are publicly discussed at the RAC's quarterly public meetings. OBA also convenes gene transfer safety symposia and policy conferences to provide a public forum for scientific experts to discuss emerging issues in the field. This transparent system of review promotes the rapid exchange of important scientific information and dissemination of data. The goal is to optimize the conduct of individual research protocols and to advance gene transfer research generally. This process has fostered the development of retroviral, lentiviral, and adeno-associated viral vector mediated gene delivery.
