Experts Tentatively OK Human Gene Editing, But With Strict Rules

Experts comprising a panel formed by the U.S. National Academies of Sciences, Engineering and Medicine have tentatively recommended that human gene editing be allowed to proceed in very limited cases where individuals are at risk of inheriting severe diseases that cannot be prevented by any other means. These edited genes would be passed on to future generations in due time, potentially removing defective genes from entire blood lines. That ample benefit, however, may not be adequate enough to quell concerns.

Human gene editing has many critics, with topics of concern including the potential future creation of so-called designer babies, as well as concerns that gene editing could be used for nefarious purposes, that it is unethical to deliberately alter the genes of a future human, and that introducing these altered genes into blood lines could cause unforeseen future issues.

Despite these concerns, gene editing also holds ample promise. Terrible, fatal or life-altering diseases inheritable from one person to the next could potentially be eradicated, allowing humans to live healthy, happy lives. Other benefits may include decreased societal burden and lowered global healthcare costs. Will such meddling ultimately destroy human evolution, though? That's yet to be seen.

There are two types of gene editing at play here, one that is non-heritable and one that is heritable. Non-heritable gene editing is, in definition, limited to one individual who will be unable to pass those altered genes onto offspring. Heritable gene editing, however, will persist in a blood line. We've already seen trials of non-heritable human gene editing go live.

The big focus now is germline genome editing — that is, the editing of genes that will cause changes that can then be inherited by offspring. With such editing, couples at risk of passing on serious medical conditions may be given a way to have their own biological children sans the risk. Such gene editing is yet to undergo actual human trials, however.

Many countries, including the U.S., currently have a ban prohibiting heritable germline editing in humans, something considered necessary until all issues can be addressed. The aforementioned committee has tentatively recommended that such gene editing be authorized only if certain strict requirements are met. First and foremost, such editing should only be allowed if there is no other 'reasonable' alternative.

As well, editing can only be done to genes that have been shown to cause serious medical conditions, and only following the acquisition of credible data on both potential benefits and risks. The committee also recommends that any clinical trials proceeding with heritable gene editing be subjected to 'ongoing, rigorous oversight,' as well as 'continued reassessment' on benefits and risks.

The committee also recommends, among other things, that ongoing and widespread input from the public be factored into any trials or decisions regarding heritable gene editing. About that, the Academies says, "Funding of genome editing research should include support to study the socio-political, ethical, and legal aspects and evaluate efforts to build public communication and engagement on these issues."

Of course, the committee's favorable attitude toward such gene editing does not mean such endeavors have been authorized or that they will be initiated at any time in the near future. Rather, the experts' newly published report — more than 240 pages long — covers all the bases and looks into all aspects of the matter, ultimately stating:

Heritable germline genome editing trials must be approached with caution, but caution does not mean they must be prohibited.

If the technical challenges are overcome and potential benefits are reasonable in light of the risks, clinical trials could be initiated, if limited to only the most compelling circumstances, are subject to a comprehensive oversight framework that would protect the research subjects and their descendants; and have sufficient safeguards to protect against inappropriate expansion to uses that are less compelling or less well understood.

The committee says that 'at this time,' it does not recommend agencies authorize gene editing of either variety 'for purposes other than treatment or prevention of disease or disability.' You can read the full report here.