Gene editing approach opens potential new route to Alzheimer's treatment
· Medical Xpressedited by Sadie Harley, reviewed by Robert Egan
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Dr. Kevin Kemp and his research team at the University of Bristol have converted the high-risk Alzheimer's gene APOE4 into the lower-risk APOE3 gene.
By targeting the gene, they hope the therapy could prevent or slow Alzheimer's in people who carry APOE4, which is found in around 1 in 4 individuals.
Kemp's research used human immune blood cells from people living with Alzheimer's disease who also carried APOE4. Using human cells allowed the researchers to observe responses as close to real-life human responses as possible.
The approach showed little impact on the rest of the cells' DNA, suggesting the therapy could be safe with minimal unwanted effects.
The results suggest the experimental gene therapy could be suitable for clinical use. It would be administered through the bloodstream, bypassing the blood-brain barrier and targeting the whole brain. The barrier has partially or completely prevented some Alzheimer's treatments from reaching all parts of the brain, making them less effective.
Kemp, an associate professor in the School of Psychology and Neuroscience at the University of Bristol, said, "Our research is still at an early stage, but we're really excited by the results we've seen so far. By targeting the high-risk APOE4 gene, we hope to tackle one of the key risk factors for Alzheimer's and ultimately develop a new treatment for the disease."
Liberty Harrison, CEO of BRACE Dementia Research, added, "We welcome this exciting development from Kemp. Therapies that can reduce or prevent APOE4 from contributing to the onset of Alzheimer's represent a major unmet clinical need and are urgently required. BRACE is a small charity, and we are proud of the impact we have had on this crucial area of research."
Key medical concepts
APOE*4 AlleleAlzheimer's DiseaseGene TherapyBlood-Brain Barrier
Clinical categories
NeurologyClinical geneticsHealthy aging Provided by University of Bristol Who's behind this story?
Sadie Harley
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