Study uses brain's glymphatic system to deliver gene therapy across blood-brain barrier
A new study presents a gene therapy strategy that leverages the brain's glymphatic transport system to deliver engineered adeno-associated viruses (AAVs) throughout the brain. This approach overcomes two key challenges in neurological medicine: crossing the blood-brain barrier and minimizing off-target effects elsewhere in the body. The platform could enable new treatments for multiple sclerosis, Huntington's disease, and rare childhood white matter disorders.
Key quotes
The approach addresses two major challenges in neurological medicine-reaching therapeutic targets behind the blood-brain barrier and limiting unwanted effects elsewhere in the body
The platform pairs specially engineered adeno-associated viruses (AAVs) with a delivery strategy
The approach could pave the way for new treatments for diseases including multiple sclerosis, Huntington's disease, and rare childhood white matter disorders
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