Gene Therapy May Prevent or Treat Alzheimer’s Disease

Although still in the early testing stages, researchers have found a potential new treatment for Alzheimer’s disease. By using gene therapy, scientists believe they could prevent or at least stop symptoms. The findings are in a paper that was just published in the journal Proceedings of the National Academy of Sciences.

The degeneration of brain cells in Alzheimer’s disease is due largely in part to amyloid plaques. The sticky protein build-up happens when amyloid proteins fold and divide improperly. The main component of these protein clumps are amyloid-beta peptides. Preventing these proteins from forming may help prevent the death of brain cells.

In research funded by the Alzheimer’s Research UK and the European Research Council, scientists from Imperial College London studied a gene called PGC-1α. Previous research had suggested that the gene could prevent the formation of dangerous amyloid-beta peptides. The team used a lentivirus, a harmless virus used in gene therapy, to inject the PGC-1α gene into mice. They injected the virus into the parts of the brain most vulnerable to Alzheimer’s, the hippocampus and cortex. The mice were treated when they had early signs of the disease but had not yet formed amyloid plaques.

The treated mice formed no or very few amyloid plaques after four months while the control group had formed many plaques. The mice who had received gene therapy behaved just like healthy mice and showed no symptoms or loss of brain cells. The treated mice also had less glial cells, which contribute to brain damage in Alzheimer’s disease. The treatment appeared effective, at least in the early stages of the disease.

The team’s results suggest that therapies utilizing PGC-1α may be able to prevent Alzheimer’s disease if the patient is treated early. Injecting the gene through a virus vector into mice prevented the build-up of amyloid plaques. Human trials are still far off but this provides new hope for the development of a treatment for a currently incurable disease.


Katsouri et al. PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model. PNAS (2016).

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