Novel gene-delivery vehicle shows promise for treating brain disorders

May 16 2024Broad Institute of MIT and Harvard Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients.

The scientists add that their new AAV could be a better option for treating neurodevelopmental disorders caused by mutations in a single gene such as Rett syndrome or SHANK3 deficiency; lysosomal storage diseases like GBA1deficiency; and neurodegenerative diseases such as Huntington's disease, prion disease, Friedreich's ataxia, and single-gene forms of ALS and Parkinson's disease.

Related StoriesTo find a delivery vehicle with a greater chance of reaching the brain in people, Deverman's team switched to a different approach. They used a method they published last year, which involves screening a library of AAVs in a test tube for ones that bind to a specific human protein. Then they test the most promising candidates in cells and mice that have been modified to express the protein.

"We've learned a lot from in vivo screens but it has been tough finding AAVs that worked this well across species," added Qin Huang, a co-first author on the study and a senior research scientist in Deverman's lab who helped develop the screening method to find AAVs that bind specific protein targets. "Finding one that works using a human receptor is a big step forward.

In work led by research scientist Jason Wu, Deverman's team also used the AAVs to deliver healthy copies of the human GBA1 gene, which is mutated in several neurological conditions. The new AAVs delivered 30 times more copies of the GBA1 gene than AAV9 in mice and were delivered throughout the brain.

Source: Healthcare Press (healthcarepress.net)

Gene Gene Therapy Blood Capsid Central Nervous System Genetic Membrane Nervous System Neurons Parkinson's Disease Prion Prion Disease Protein Receptor Research

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