Scientists find a possible gene therapy for Alzheimer’s disease

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gene therapy for Alzheimer’s disease

In a recent study conducted by Imperial College London, researchers find a way to prevent Alzheimer’s disease in mice by using a virus to deliver a specific gene into the brain. The finding is newly published in PNAS.

The gene, PPARγ-coactivator-1α (PGC-1α), is a transcriptional regulator of genes involved in energy metabolism.

In the previous studies, researchers have observed that PGC-1α could decrease the Aβ pathology in cell culture, and its levels were reduced in Alzheimer’s disease brains.

Aβ, or Amyloid beta, is crucially involved in Alzheimer’s disease and usually found in the brains of Alzheimer patients.

To determine the gene’s potential therapeutic role, researchers injected PGC-1α into specific brain areas of mice that had Alzheimer’s disease.

The result showed that 4 months after gene injection, mice treated with PGC-1α showed improved spatial and recognition memory.

In these mice’s brain, the expression of the main enzyme involved in Aβ production were strongly reduced, which means most neurons in the brain survived.

Based on the finding, researchers suggest that PGC-1α delivery shows promising therapeutic value in Alzheimer’s disease.

In addition, injections of the gene will be most beneficial in the early stages of the disease, when the first symptoms appear.

This study has significant implications for public health. Alzheimer’s disease is the most common form of dementia, affecting up to 70% of all people with dementia.

In most people with Alzheimer’s, symptoms first appear in their mid-60s. Estimates vary, but experts suggest that more than 5 million Americans may have Alzheimer’s.

Although currently there is no treatment able to halt the progression of damage in Alzheimer’s, this research is a promising step on the road towards developing treatments for this devastating condition.

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Citation: Katsouri L, et al. (2016). PPAR-gamma-coactivator-1-alpha gene transfer reduces neuronal loss and amyloid-beta generation by beta-secretase in and Alzheimer’s disease model. PNAS, published online. DOI: 10.1073/pnas.1606171113.
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