Sci­en­tists re­pair gene de­fect in em­bryo

China Daily (Hong Kong) - - FRONT PAGE -

WASH­ING­TON — In a first, re­searchers safely re­paired a dis­ease-caus­ing gene in hu­man em­bryos, tar­get­ing a heart de­fect best known for killing young ath­letes — a big step to­ward some day pre­vent­ing a list of in­her­ited dis­eases.

In a sur­pris­ing dis­cov­ery, the re­search team led by Ore­gon Health & Sci­ence Uni­ver­sity re­ported on Wed­nes­day that em­bryos can help fix them­selves if sci­en­tists jump­start the process early enough.

It was lab­o­ra­tory re­search only, nowhere near ready to be tried in a preg­nancy. But it sug­gests that sci­en­tists might be able to al­ter DNA in a way that pro­tects not just one baby from a dis­ease that runs in the fam­ily, but his or her off­spring as well. And that raises eth­i­cal ques­tions.

The new method uses the ge­need­it­ing tool CRISPR to tar­get a mu­ta­tion in nu­clear DNA that causes hy­per­trophic cardiomyopathy, a com­mon ge­netic heart con­di­tion that can cause sud­den car­diac death and heart fail­ure.

The dis­ease af­fects an es­ti­mated one in 500 peo­ple and can lead to heart fail­ure and sud­den death.

Re­searchers worked with healthy do­nated hu­man oocytes, or egg cells, in ovaries that form an ovum and sperm car­ry­ing the ge­netic mu­ta­tion that causes cardiomyopathy. Em­bryos cre­ated in this study were used to an­swer pre­clin­i­cal ques­tions about safety and ef­fec­tive­ness.

“Ev­ery gen­er­a­tion would carry on this re­pair be­cause we’ve re­moved the dis­ease­caus­ing gene vari­ant from that fam­ily’s lin­eage,” said the se­nior author of the re­port, Shoukhrat Mi­tal­ipov, who di­rects the Cen­ter for Em­bry­onic Cell and Gene Ther­apy at the uni­ver­sity.

CRISPR stands for clus­tered reg­u­larly in­ter­spaced short palin­dromic re­peats. It holds prom­ise for cor­rect­ing mu­ta­tions to pre­vent other ge­netic dis­eases.

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