Human gene editing research gets green light

WASHINGTON, D.C. — Human gene-editing research, even on embryos, is needed and should go ahead, with one major caveat: No pregnancies can result, leaders of an international summit on the topic said December 3.

In recent years, scientists have devised increasingly precise molecular scissors for cutting and pasting DNA. These tools, especially the guided scissors known as CRISPR/Cas9, have become so cheap and easy to use that it may be possible to use them to correct genetic diseases.

Many see the technology as a medical boon; others, though, say that the prospect of designer babies and tinkering with the DNA of future generations should be out of bounds (SN: 5/30/15, p. 16). The U.S. National Academies of Sciences and Medicine, the Chinese Academy of Sciences and the United Kingdom’s Royal Society convened the summit to discuss the state of the science as well as ethical, legal and regulatory considerations surrounding gene-editing technology.

Gene editing of human body, or somatic, cells, which do not pass genetic information to future generations, is already in clinical trials. Most of those studies have involved older technologies and cells that were edited outside the body and then given to a patient later, such as a baby with leukemia treated with edited immune cells (SN: 12/12/15, p. 7).

A company called Sangamo BioSciences announced December 1 that clinical trials using gene editing to replace a broken gene in adult hemophiliacs could begin next year. Such research could continue and would fall under current regulations for gene therapy, the 12-member organizing committee of the International Summit on Human Gene Editing said in statement.

But moral, ethical and safety concerns would make it “irresponsible” to proceed with clinical studies in germline cells — eggs, sperm, embryos and other cells that transmit DNA to future generations, the statement added. That doesn’t mean all germ cell editing would be off-limits. Researchers who edit embryos or other germ cells in labs would not be doing germline editing if the resulting embryos are not implanted in the uterus for reproductive purposes, said committee chairman David Baltimore of Caltech.

The scientists purposely did not call their statement a ban or even a moratorium. Instead, the recommendations should be revisited on a regular basis as research advances and societal opinions evolve. The panel also called for an ongoing forum to discuss human germline editing.

Recommendations from the scientists are not legally binding, but peer pressure could be an effective deterrent. For instance, researchers who violate agreements might not be able to get their work published or could lose funding. Scientists also must still follow their individual countries’ laws and regulations on working with embryos. In the United States, such work is not banned, but researchers cannot get government funds to do it.

A study conducted by a separate committee of scientists commissioned by the science academies will produce a report on the advisability of germline editing, expected by the end of 2016.

Editor’s note: The headline of this story was edited on December 7 to clarify, as noted elsewhere, that the recommendations endorsed germ cell gene editing for research purposes only.