Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2015;15(12):25-9.
doi: 10.1080/15265161.2015.1104160.

Human Germline CRISPR-Cas Modification: Toward a Regulatory Framework

Niklaus H Evitt  1 , Shamik Mascharak  1 , Russ B Altman  1
Affiliations

Human Germline CRISPR-Cas Modification: Toward a Regulatory Framework

Niklaus H Evitt et al. Am J Bioeth. 2015.

Abstract

CRISPR germline editing therapies (CGETs) hold unprecedented potential to eradicate hereditary disorders. However, the prospect of altering the human germline has sparked a debate over the safety, efficacy, and morality of CGETs, triggering a funding moratorium by the NIH. There is an urgent need for practical paths for the evaluation of these capabilities. We propose a model regulatory framework for CGET research, clinical development, and distribution. Our model takes advantage of existing legal and regulatory institutions but adds elevated scrutiny at each stage of CGET development to accommodate the unique technical and ethical challenges posed by germline editing.

Keywords: CRISPR-Cas; clinical ethics; gene editing; gene therapy; genetic disease; germline modification.

PubMed Disclaimer

Figures

Figure 1.
Figure 1.
A model regulatory pathway for developing CRISPR germline editing therapies.
See this image and copyright information in PMC

Comment in

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Baltimore D., Berg P., Botchan M., Carroll D., Charo R. A., Church G., Corn J. E., Daley G. Q., Doudna J. A., Fenner M., Greely H. T., Jinek M., Martin G. S., Penhoet E., Puck J., Sternberg S. H., Weissman J. S., Yamamoto K. R. A prudent path forward for genomic engineering and germline gene modification. Science. 2015;348(6230):36–38. - PMC - PubMed
    1. Bosley K. S., Botchan M., Bredenoord A. L., Carroll D., Charo R. A., Charpentier E., Cohen R., Corn J., Doudna J., Feng G., Greely H. T., Isasi R., Ji W., Kim J. S., Knoppers B., Lanphier E., Li J., Lovell-Badge R., Martin G. S., Moreno J., Naldini L., Pera M., Perry A. C., Venter J. C., Zhang F., Zhou Q. CRISPR germline engineering—The community speaks. Nature Biotechnology. 2015;33(5):478–86. - PubMed
    1. Collins F. S. Statement on NIH funding of research using gene-editing technologies in human embryos. National Institutes of Health, Department of Health and Human Services; 2015.
    1. Esvelt K. M., Smidler A. L., Catteruccia F., Church G. M. Concerning RNA-guided gene drives for the alteration of wild populations. Elife. 2014:e03401. doi: 10.7554/eLife.03401. - DOI - PMC - PubMed
    1. Food and Drug Administration FDA guidance for industry: Gene therapy clinical trials—Observing subjects for delayed adverse events 2006

MeSH terms

LinkOut - more resources