title: Human Germline CRISPR-Cas Modification: Toward a Regulatory Framework.
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.
authors.affiliation: a Stanford University.
authors.forename: Niklaus H
authors.initials: NH
authors.lastname: Evitt
authors.affiliation: a Stanford University.
authors.forename: Shamik
authors.initials: S
authors.lastname: Mascharak
authors.affiliation: a Stanford University.
authors.forename: Russ B
authors.initials: RB
authors.lastname: Altman
mesh_terms: D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D030342:Genetic Diseases, Inborn / Q000175:diagnosis / Q000235:genetics* / Q000517:prevention & control*; D005818:Genetic Engineering / Q000941:ethics / Q000331:legislation & jurisprudence*; D036281:Genetic Research / Q000941:ethics / Q000331:legislation & jurisprudence*; D018095:Germ-Line Mutation; D017408:Guidelines as Topic; D006801:Humans; D011296:Prenatal Diagnosis / Q000941:ethics / Q000379:methods / Q000639:trends; D011640:Public Policy / Q000331:legislation & jurisprudence / Q000639:trends; D014481:United States
keywords: CRISPR-Cas; clinical ethics; gene editing; gene therapy; genetic disease; germline modification