Hit­ting land­mark mile­stone, In­tel­lia shows CRISPR can ed­it genes di­rect­ly in pa­tients

In a land­mark study, re­searchers suc­cess­ful­ly used CRISPR to di­rect­ly ed­it DNA in hu­mans, a mile­stone that could pave the way for treat­ments for scores of se­ri­ous ge­net­ic and non-ge­net­ic dis­eases.

In­tel­lia, a biotech co-found­ed by 2020 No­bel lau­re­ate Jen­nifer Doud­na, used CRISPR/Cas9 to cut a gene out of the liv­er cells of pa­tients with AT­TR amy­loi­do­sis, a dead­ly dis­ease where a mis­shapen pro­tein called TTR builds up and dam­ages or­gans through­out the body. Pa­tients who re­ceived a high dose of the ther­a­py saw their pro­tein lev­els fall be­tween 80% and 96%, in­di­cat­ing the ther­a­py per­ma­nent­ly cut the genome where de­sired.

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