Gene therapy may hold promise for preventing Alzheimer’s progression

Novel method targeting PGC1-α shows promise in mouse study

A new study from researchers at Imperial College London (UK) has demonstrated the efficacy of a gene therapy for prevention of Alzheimer’s disease development in mice.

The gene employed in this new approach is PGC1-α, a regulator of energy metabolism. PGC1-α was delivered via injection of a lentivirus vector into specific brain regions in mice known to be susceptible to Alzheimer’s.

Animals were treated while in the early stages of Alzheimer’s disease, prior to the development of amyloid plaques. At four months post-treatment, mice who had received the gene were observed to have very few amyloid plaques when compared with untreated controls. This finding supported the group’s previous work suggesting that PGC1-α may prevent amyloid-β peptide formation. Treated mice also demonstrated no loss of neurons in the hippocampus, a reduction in glial cells and memory test performance equivalent to healthy comparison mice.

“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,” summarized senior author Magdalena Sastre (Imperial College London).

Written by Hannah Wilson

Source: Imperial College London News www3.imperial.ac.uk/newsandeventspggrp/imperialcollege/newssummary/news_10-10-2016-16-17-41