💰 A Promising Trial Targets a Genetic Risk for Alzheimer’s

TL;DR

A Promising Trial Targets a Genetic Risk for Alzheimer’s Preliminary results offer hope that gene therapy can protect people with a version of the brain disease driven by a particular gene variant.But the preliminary results, announced Friday during the Clinical Trials on Alzheimer’s Disease conference, showed that proteins from the added gene appeared in the patients’ spinal fluid, and levels in the brain of two markers of Alzheimer’s disease, tau and amyloid, fell.Treatment of another five patients at a higher dose is underway, and the work, initially funded by the nonprofit Alzheimer’s Drug Discovery Foundation, is supported by Lexeo Therapeutics, a fledgling company founded by Dr. Ronald Crystal, who is also chairman of the department of genetic medicine at Weill Cornell Medicine in New York.That leads to the idea that if gene therapy floods the brain with APOE2, converting the brain milieu of a person with two APOE4 variants to one that resembles that of a person with one APOE4 and one APOE2, it could possibly slice Alzheimer’s risk in half.Recruitment to try the technique has been slow, said Dr. Sam Gandy, professor of Alzheimer’s disease research at Mount Sinai in New York, who was one of the study investigators."

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