3-D illustration of an AAV.
Scientists at the Broad Institute of MIT and Harvard have developed a new method to deliver gene therapies across the blood-brain barrier which is a very important advancement for treating neurological and mental disorders. They used an adeno-associated virus (AAV) made to bind to the human transferrin receptor (TfR1) so it can let the gene therapy reach brain cells more effectively than other techniques out there.
This treatment uses the blood-brain barrier and acts as a filter to protect the brain, which could also lead to blocking many treatments from reaching brain cells. The researchers changed the AAV to latch onto a protein called the human transferrin receptor (TfR1), which then helps it slip past this barrier. Tests on human cells and mice with human TfR1 protein showed that this new AAV, called BI-hTFR1, was far more effective than current AAVs. It was shown to be more efficient in crossing the barrier and delivering genetic material right where it was needed in the brain.
One test involved delivering the GBA1 gene, which is connected to diseases like Parkinson’s and Gaucher disease. This new AAV delivered GBA1 up to 30 times more effectively than existing methods. Though it’s still early days and more testing is needed, in my opinion, this technique could lead to better, safer treatments for brain disorders. This gives new hope to millions who suffer from conditions that currently have limited options.
Reference:
https://www.nimh.nih.gov/news/science-news/2024/new-gene-delivery-method-paves-the-way-for-advanced-brain-therapies
Additional:
https://www.doi.org/10.1126/science.adm8386
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