Tags: Alzheimer's/Dementia | gene | therapy | treat | Alzheimers | disease | brain

Can Gene Therapy Treat Alzheimer's Disease?

Can Gene Therapy Treat Alzheimer's Disease?

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By    |   Tuesday, 11 October 2016 02:17 PM

Scientists have prevented the development of Alzheimer's in mice by using a modified virus to deliver a specific gene to brain cells.

Researchers from Imperial College London say that their early-stage findings open new paths for potential treatments that may prevent or stop the mind-robbing disease in its tracks.

Previous studies by the researcher suggest that a gene called PGC1-alpha may prevent the formation of a protein called amyloid-beta peptide in cells in the lab.

Amyloid-beta peptide is the main component of amyloid plaques, the sticky clumps of protein found in the brains of people with Alzheimer's disease. These plaques are thought to trigger the death of brain cells.

There is no cure, although current drugs can help treat the symptoms of the disease.
"Although these findings are very early they suggest this gene therapy may have potential therapeutic use for patients," said senior author Dr. Magdalena Sastre.

"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."

The modified virus used in the experiments was a lentivirus vector, which is commonly used in gene therapy, according to study co-author Nicholas Mazarakis.

"Scientists harness the way lentivirus infects cells to produce a modified version of the virus, that delivers genes into specific cells," said Mazarakis.

"It is being used in experiments to treat a range of conditions from arthritis to cancer. We have previously successfully used the lentivirus vector in clinical trials to deliver genes into the brains of Parkinson's disease patients."

In the new study, the team injected the virus, containing the gene PGC-1-alpha, into two areas of the brain in mice—the hippocampus and the cortex—which are susceptible to Alzheimer's and are the first regions to develop amyloid plaques.

Damage to the hippocampus affects short-term memory, and leads to a person forgetting recent events, such as a conversation or what they ate for breakfast.

The hippocampus is also responsible for the sense of direction, and damage results in a person becoming lost on familiar journeys, such as driving home from shopping.

The cortex is responsible for long-term memory, reasoning, thinking, and mood. Damage can trigger symptoms such as depression, struggling to work out how much money to give at a checkout, how to get dressed, or how to cook a familiar recipe.

The animals in the study were in the early stage of Alzheimer's, and had not yet developed amyloid plaques.

After four months, the team found that mice who received the gene had very few amyloid plaques, compared with the untreated mice, who had multiple plaques in their brain.

In addition, the treated mice performed as well in memory tests as healthy mice.

The researchers also discovered there was no loss of brain cells in the hippocampus of the mice who received the gene treatment.

In addition, the treated mice had a reduction in the number of glial cells, which can release toxic inflammatory chemicals that cause further cell damage in Alzheimer's victims.

Dr. Sastre noted that other studies from different institutions suggest physical exercise and the compound resveratrol, found in red wine, may increase levels of PGC-1-alpha protein.

However, resveratrol appears to have benefits as a pill, rather than in wine, since the alcohol counteracts any benefit.

Dr. David Reynolds, Chief Scientific Officer at Alzheimer's Research UK, said: "There are currently no treatments able to halt the progression of damage in Alzheimer's, so studies like this are important for highlighting new and innovative approaches to take us towards that goal."

"The findings support PGC-1-alpha as a potential target for the development of new medicines, which is a promising step on the road towards developing treatments for this devastating condition."

The study was published in the journal Proceedings of the National Academy of Sciences.
Other research has pointed to methods using proteins to reduce amyloid plaques.

Researchers at the Salk Institute have alleviated characteristics of Alzheimer's disease by boosting a protein in the brains of mice bred to develop the condition, according to a study published in Scientific Reports.

The protein, called neuregulin-1, has many forms and functions across the brain and is already a potential target for brain disorders such as Parkinson's disease, amyotrophic lateral sclerosis and schizophrenia.

Researchers raised the levels one of two forms of neuregulin-1 in the hippocampus, and both forms of the protein seemed to improve performance on a test of spatial memory in the mice.

In addition, the levels of cellular markers of disease, including the levels of amyloid beta and plaques, were noticeably lower in mice with more neuregulin-1 compared to controls.

The study suggests that neuregulin-1 breaks up plaques by raising levels of an enzyme called neprilysin, which has been shown to degrade amyloid-beta.

According to the Alzheimer's Association, 5.4 million Americans are living with Alzheimer's, and the number of sufferers may triple by 2050.


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Scientists have prevented the development of Alzheimer's in mice by using a modified virus to deliver a specific gene to brain cells. Researchers from Imperial College London say that their early-stage findings open new paths for potential treatments that may prevent or...
gene, therapy, treat, Alzheimers, disease, brain, protein
Tuesday, 11 October 2016 02:17 PM
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