Cor­nell re­searchers to launch ear­ly tri­al of gene ther­a­py aimed — ul­ti­mate­ly — at pre­vent­ing Alzheimer's

The no­to­ri­ous dif­fi­cul­ty — if not im­pos­si­bil­i­ty — of re­vers­ing or slow­ing the pro­gres­sion of Alzheimer’s has pushed re­searchers to study much ear­li­er stages of the dis­ease, and the po­ten­tial of treat­ing pa­tients be­fore they show signs of brain dam­age. But can they go even ear­li­er than that — by tak­ing a pre­ven­tion ap­proach based on a ge­net­ic dri­ver of the dis­ease?

Doc­tors at Weill Cor­nell Med­i­cine are giv­ing it a try with a gene ther­a­py de­signed to flood the brains of high-risk pa­tients with a low-risk ver­sion of the APOE gene, there­by knock­ing down their risk of get­ting Alzheimer’s to just av­er­age. In three months — pend­ing man­u­fac­tur­ing clear­ance — they will be­gin a 15-per­son tri­al to test if their in­fu­sion can in­deed lead to the right mix of genes (and sub­se­quent pro­duc­tion of pro­teins) in the brain.

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