Article
Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B.
Human gene therapy - 1 Jan 2020
Batista Ana Rita, King Oliver D, Reardon Christopher P, Davis Crystal, Shankaracharya, Philip Vivek, Gray-Edwards Heather, Aronin Neil, Lutz Cathleen, Landers John, Sena-Esteves Miguel
Abstract excerpt
Adeno-associated virus (AAV) gene therapy for neurological diseases was revolutionized by the discovery that AAV9 crosses the blood-brain barrier (BBB) after systemic administration. Transformative results have been documented in various inherited diseases, but overall neuronal transduction efficiency is relatively low. The recent development of AAV-PHP.B with ∼60-fold higher efficiency than AAV9 in transducing...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
