Sci­en­tists edit genes of hu­man em­bryos for first time in U.S.

The Maui News - - FRONT PAGE - By MARILYNN MARCHIONE The As­so­ci­ated Press

For the first time in the United States, sci­en­tists have edited the genes of hu­man em­bryos, a con­tro­ver­sial step to­ward some­day help­ing ba­bies avoid in­her­ited dis­eases.

The ex­per­i­ment was just an ex­er­cise in science — the em­bryos were not al­lowed to de­velop for more than a few days and were never in­tended to be im­planted into a womb, ac­cord­ing to MIT Tech­nol­ogy Re­view, which first re­ported the news.

Of­fi­cials at Ore­gon Health & Science Univer­sity con­firmed Thurs­day that the work took place there and said re­sults would be pub­lished in a jour­nal soon. It is thought to be the first such work in the U.S.; pre­vi­ous ex­per­i­ments like this have been re­ported from China. How many em­bryos were cre­ated and edited in the ex­per­i­ments has not been re­vealed.

The Ore­gon sci­en­tists re­port­edly used a tech­nique called CRISPR, which al­lows spe­cific sec­tions of DNA to be al­tered or re­placed. It’s like us­ing a molec­u­lar scis­sors to cut and paste DNA, and is much more pre­cise than some types of gene ther­apy that can­not en­sure that de­sired changes will take place ex­actly where and as in­tended. With gene edit­ing, th­ese so-called “germline” changes are per­ma­nent and would be passed down to any off­spring.

The ap­proach holds great po­ten­tial to avoid many ge­netic dis­eases, but has raised fears of “de­signer ba­bies” if done for less lofty rea­sons, such as pro­duc­ing de­sir­able traits.

Last year, Bri­tain said some of its sci­en­tists could edit em­bryo genes to bet­ter un­der­stand hu­man de­vel­op­ment.

And ear­lier this year in the U.S., the Na­tional Academy of Sci­ences and Na­tional Academy of Medicine said in a re­port that al­ter­ing the genes of em­bryos might be OK if done un­der strict cri­te­ria and aimed at pre­vent­ing se­ri­ous dis­ease.

“This is the kind of re­search that the re­port dis­cussed,” Univer­sity of Wis­con­sin-Madi­son bioethi­cist R. Alta Charo said of the news of Ore­gon’s work. She co-led the Na­tional Acad­e­mies panel but was not com­ment­ing on its be­half Thurs­day.

“This was purely lab­o­ra­to­ry­based work that is in­cred­i­bly valu­able for help­ing us un­der­stand how one might make th­ese germline changes in a way that is pre­cise and safe. But it’s only a first step,” she said.

“We still have reg­u­la­tory bar­ri­ers in the United States to ever try­ing this to achieve a preg­nancy. The pub­lic has plenty of time” to weigh in on whether that should oc­cur, she said. “Any such ex­per­i­ment aimed at a preg­nancy would need FDA approval, and the agency is cur­rently not al­lowed to even con­sider such a re­quest” be­cause of limits set by Congress.

One prom­i­nent ge­net­ics ex­pert, Dr. Eric Topol, di­rec­tor of the Scripps Trans­la­tional Science In­sti­tute in La Jolla, Calif., said gene edit­ing of em­bryos is “an un­stop­pable, in­evitable science, and this is more proof it can be done.”

Ex­per­i­ments are in the works now in the U.S. us­ing gene-edited cells to try to treat peo­ple with var­i­ous dis­eases, but “in order to re­ally have a cure, you want to get this at the em­bryo stage,” he said. “If it isn’t done in this coun­try, it will be done else­where.”

There are other ways that some par­ents who know they carry a prob­lem gene can avoid pass­ing it to their chil­dren, he added. They can cre­ate em­bryos through in vitro fer­til­iza­tion, screen them in the lab and im­plant only ones free of the de­fect.

Dr. Robert C. Green, a med­i­cal ge­neti­cist at Har­vard Med­i­cal School, said the prospect of edit­ing em­bryos to avoid dis­ease “is in­evitable and ex­cit­ing,” and that “with proper con­trols in place, it’s go­ing to lead to huge ad­vances in hu­man health.”

The need for it is clear, he added: “Our re­search has sug­gested that there are far more dis­ease-as­so­ci­ated mu­ta­tions in the gen­eral pub­lic than was pre­vi­ously sus­pected.”

Hank Greely, di­rec­tor of Stan­ford Univer­sity’s Center for Law and the Bio­sciences, called CRISPR “the most ex­cit­ing thing I’ve seen in bi­ol­ogy in the 25 years I’ve been watch­ing it,” with tremen­dous pos­si­bil­i­ties to aid hu­man health.

“Every­body should calm down” be­cause this is just one of many steps ad­vanc­ing the science, and there are reg­u­la­tory safe­guards al­ready in place. “We’ve got time to do it care­fully,” he said.

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