Gene therapy could eventually be used to treat Alzheimer’s disease, British scientists have announced.
The London-based researchers say they have successfully prevented the disease in mice using a gene therapy.
The treatment involved using a viral vector to deliver the PGC1-alpha gene following earlier work that suggested it might prevent the formation of amyloid-beta peptide.
During the research the therapy was delivered to the hippocampus and the cortex.
The researchers report that after four months treated mice had very few amyloid plaques compared with untreated animals.
The treated animals also seemed to experience no loss of cells in the hippocampus – and their memories appeared as good as those of healthy animals.
The findings have been published in the Proceedings of the National Academy of Sciences.
Researcher Dr Magdalena Sastre, of Imperial College, London, said: “Although these findings are very early they suggest this gene therapy may have potential therapeutic use for patients. There are many hurdles to overcome, and at the moment the only way to deliver the gene is via an injection directly into the brain.
"However this proof of concept study shows this approach warrants further investigation.”
Alzheimer’s Research UK said the findings were "promising."
Chief scientific officer Dr David Reynolds said: "This research sets a foundation for exploring gene therapy as a treatment strategy for Alzheimer’s disease, but further studies are needed to establish whether gene therapy would be safe, effective and practical to use in people with the disease."
L. Katsouri et al. PPAR-gamma-coactivator-1-alpha gene transfer reduces neuronal loss and amyloid-beta generation by beta-secretase in and Alzheimer’s disease model PNAS 10 October 2016
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