Methods and compositions for delivery of agents across the blood-brain barrier
AssigneeThe Brigham and Women's Hospital, Inc.
InventorFengfeng Bei
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Abstract
Sequences that enhance permeation of agents into cells and/or across the blood brain barrier, compositions comprising the sequences, and methods of use thereof.
Claims36
A method of delivering a transgene to a subject, the method comprising administering to the subject an adeno-associated virus (AAV) vector comprising (i) a transgene, and (ii) an AAV capsid comprising a peptide insert of up to 21 amino acids, wherein the peptide insert comprises 5-7 contiguous amino acids of TVSALFK (SEQ ID NO:
- 8).
A method of delivering a non-coding RNA to a subject, the method comprising administering to the subject an adeno-associated virus (AAV) vector comprising (i) a non-coding RNA, and (ii) an AAV capsid comprising a peptide insert of up to 21 amino acids, wherein the peptide insert comprises 5-7 contiguous amino acids of TVSALFK (SEQ ID NO:
- 8).
A method of treating a disorder of the brain or central nervous system in a subject, the method comprising administering to the subject an AAV vector comprising (i) a non-coding RNA or a transgene encoding a therapeutic agent, and (ii) an AAV capsid comprising a peptide insert of up to 21 amino acids, wherein the peptide insert comprises 5-7 contiguous amino acids of TVSALFK (SEQ ID NO:
- 8), thereby treating the disorder.
Dependencies are inferred from the claim text.