In a groundbreaking development for neuroscience and medicine, researchers at the National Institute of Health have announced promising results from Phase II clinical trials of an innovative gene therapy targeting Alzheimer’s disease. The treatment, which uses CRISPR-based technology to modify specific genes associated with amyloid plaque formation, has shown significant cognitive improvements in 68% of trial participants.
The therapy works by targeting the APOE4 gene variant, known to increase Alzheimer’s risk, and converting it to the protective APOE2 variant. Lead researcher Dr. Jennifer Liu explained that early intervention patients showed the most dramatic improvements in memory retention and cognitive function over the 18-month trial period.
While the treatment is still years away from widespread availability, the FDA has granted it breakthrough therapy designation, potentially accelerating the approval process. Experts believe this could mark a turning point in the fight against Alzheimer’s, which currently affects over 6 million Americans. Phase III trials are scheduled to begin in early 2026.
