Gene-editing technologies, such as clustered regularly interspaced short palindromic repeats–Cas9 (CRISPR/Cas9), are feasible and effective for manipulating somatic and germline genomes, according to a report published in JAMA.1
In this perspective article, the investigators report on a new consensus statement by 11 organizations led by the American Society of Human Genetics (ASHG).2 According to the report, no reasons exist to support the prohibition of in vitro germline genome editing in human gametes and embryos. This editing, however, should be performed only following consent from donors.
Overall, this statement describes the utility of CRISPR/Cas9, among other gene-editing tools, and suggests the eventual therapeutic potential of human DNA editing as it relates to effects that can be passed to future generations.
The report also highlights the importance of public funding for gene editing research, especially with regard to studies aimed at manipulating human embryos and fetuses.
As with most research involving gene editing in humans, the organizations discussed the ethical considerations of germline gene editing. Specifically, the organizations state that human germline genome editing should only be conducted if there is a compelling medical-related rationale, evidence or literature that support its use, public transparency, and an ethical justification.
The statement from the ASHG panel notes that, at this time, using germline manipulation for therapeutic means is premature. Effects of off-target mutations and the possible effects of desired on-target modifications present safety challenges that currently make this practice not possible. Additionally, because there are a number of unanswered ethical, policy, and scientific questions, the investigators note that germline gene editing that results in human pregnancy is also inappropriate.
In addition to soliciting input from the public, the ASHG statement,“ acknowledged that a significant educational effort would be needed” before proceeding with genome editing.
1. Lyon J. Bioethics panels open door slightly to germline gene editing. JAMA. 2017;318(17):1639-1640.
2. Ormond KE, Mortlock DP, Scholes DT, et al. Human germline genome editing. Am J Hum Genet. 2017;101(2):167-176.