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Longevity Briefs: Gene therapy for Alzheimer’s disease

Posted on 26 January 2021

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Longevity briefs provides a short summary of novel research in biology, medicine, or biotechnology that caught the attention of our researchers in Oxford, due to its potential to improve our health, wellbeing, and longevity.

Why is this research important: Alzheimers is the most common form of dementia, a neurological condition which sees the degradation of cognitive ability to a senile state, eventually resulting in death.

The risk of developing Alzheimers has been heavily associated with the APOE gene.

The APOE gene consists of a combination of two of the APOE alleles, of which there are three variations: E2, E3 and E4. The combination of which decides the APOE genotype of every individual; E2/E2, E2/E3, E3/E4 etc.

Source: Queensland Brain Institute

Individuals who possess two E2 alleles, and therefore have the E2/E2 genotype, have a far lower chance of developing Alzheimer’s, whereas those who have two E4 alleles are much more likely to develop the neurodegenerative disease.

What did the researchers do: A clinical trial sponsored by Weill Medical College of Cornell University, in the US, is evaluating a novel gene therapy to treat patients who are E4/E4 and who already experiencing Alzheimer’s symptoms.

By introducing a viral vector containing complementary DNA of the E2 allele into the central nervous system of the patients, the researchers are hoping to slow down the progression of the disease.

Source: FDA

Key takeaway(s) from this research: This is a phase 1 clinical trial, which means that the data will primarily be used to determine whether the new therapy is safe. However, the data will also give us some indication of how effective the therapy has been.

The completion of the study is scheduled to be in December of this year. We will be eagerly awaiting the results!

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