Article

An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery

2023-12-22

Abstract excerpt

Developing vehicles that efficiently deliver genes throughout the human central nervous system (CNS) will broaden the range of treatable genetic diseases. We engineered an AAV capsid, BI-hTFR1, that binds human Transferrin Receptor (TfR1), a protein expressed on the blood-brain barrier (BBB). BI-hTFR1 was actively transported across a human brain endothelial cell layer and, relative to AAV9, provided 40–50 times g...

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Identifiers and source

Literature Corpus work: 0ab43e52-0aa4-5f63-8091-919f7e528b14
DOI: 10.1101/2023.12.20.572615

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