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Can we use gene therapy to treat alzheimer’s disease?

ByMatt Grant

Nov 7, 2016

There are an estimated 52,000 Alzheimer’s disease sufferers in the UK, and it can be a torment for those who are affected.

However, gene therapy may offer the world a new hope. A new study that used a modified virus to insert a beneficial gene into the brains of mice was able halt the development of the disease.

Alzheimer’s disease can have heart-wrenching consequences, both for sufferers and the friends and family supporting them.

Long and short-term memory loss often occurs, along with stark mood swings and personality changes. Symptoms are thought to be due to the buildup of sticky protein clumps known as amyloid plaques, which can lead to the death of important neurons in regions of the brain responsible for memory, reasoning and mood.

Current treatments for Alzheimer’s disease are able to dampen these symptoms, buying extra time for patients, but are unable to fully halt the progression of the disease.

Scientists found they could reduce the build-up of amyloid plaques by inserting a gene, PGC1-alpha, into mice with early stage Alzheimer’s.

The treatment was so effective that treated mice could perform as well as normal healthy mice in memory tests.

In order to insert the beneficial gene, researchers used a type of virus (called a lentivirus) as a delivery system.

This virus was able to penetrate deep into the nucleus of cells, before integrating the beneficial gene with current DNA.

Once integrated, more of the protein that helps clear the sticky amyloid plaque is produced and helps prevent the death of neurons.

If this technique could be translated to humans and combined with early diagnosis, there is the hope that the disease can be stopped before symptoms become severe.

Dr. Magdalena Sastre, senior author for the study, explained: “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.”

While this work is undeniably in the early stages of development, the same modified virus technique was shown to have some success in treating Parkinson’s patients.

This study will hopefully lay the foundation for gene therapy as a viable method of treatment for Alzheimer’s disease in the future, although it may be some time before the approach sees human trials.

Image: Gemma Evans

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