Doctors perform gene therapy for Alzheimer's disease in humans for first time

December 9, 2022  22:31

Researchers from the American company Lexeo Therapeutics announced the successful completion of the first phase of clinical trials of gene therapy for Alzheimer's disease at the Clinical Trials on Alzheimer's Disease conference, The New York Times reports.

According to numerous studies, people with the APOE4 protein gene are at greater risk of getting dementia than others, while a variation of the same gene - APOE2 - reduces that likelihood. Scientists decided that a disease characterized by cognitive problems could be cured by grafting the APOE2 gene into a person's body.

The first Alzheimer's disease gene therapy trial involved only five patients with a high genetic risk of developing Alzheimer's disease. They received very low doses of the APOE2 gene drug to evaluate its safety. The drug was injected once into the craniocervical junction, the place where the skull transitions to the neck.

The results of the experiment showed that APOE2 proteins began to be produced in the subjects' cerebrospinal fluid. This means that the therapy was successful.

Moreover, the subjects had reduced levels of tau protein and beta-amyloid, high concentrations of which are characteristic of dementia patients.

The scientists promised that they would soon begin the next stage of research.

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