In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke (NINDS), a group of researchers have created a novel adeno-associated viral (AAV) vector to address brain disorders. This vector is more effective at reaching brain cells, opening doors for the development of treatment for brain disorders such as Parkinson's, Alzheimer's, and Huntington's Disease.
Adeno-associated viral (AAV) vectors are commonly used in gene therapy to deliver necessary genetic material to the target cells in our bodies. Here, they release and supply the genetic material like they are supposed to and provide a long-term solution for the target cells.
Cutting-Edge AAV Gene Delivery for Brain Disorders
For years, the biggest challenge in using gene therapy for treating brain disorders has been the blood-brain barrier (BBB). This is a protective layer that is responsible for blocking out harmful things from the brain, ultimately also blocking out a lot of therapeutics from entering the brain. The novel AAV vector that was created by this group of scientists, named BI-hTFR, has the ability to overcome the blood-brain barrier and reach the brain. Specifically, the passing involves BI-hTFR binding to a human transferrin receptor (TfR1). In preclinical trials on mice, this novel vector successfully delivers 30 times more copies of a gene compared to a normal AAV vector.
The invention of this new technology suggests the potential for the development of minimally invasive treatments for brain disorders. Some worth-noting disorders of interest are Parkinson's, Huntington's, Alzheimer's, ALS, schizophrenia...
While further research is needed, this breakthrough paves new ways for safer, more effective, and more target treatments of brain disorders.
Learn more about the challenges of delivering gene therapy across the blood-brain barrier and how AAV capsids are changing the future of brain disorder treatments. Interview with field expert - Amy Pooler, PhD.
(Media Source: https://www.youtube.com/watch?v=sC4eCO-jKCU)
Created: August 20th, 2024
Citations:
“New Gene Delivery Method Paves the Way for Advanced Brain Therapies.” National Institute of Mental Health, U.S. Department of Health and Human Services, 15 Aug. 2024, www.nimh.nih.gov/news/science-news/2024/new-gene-delivery-method-paves-the-way-for-advanced-brain-therapies.
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