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US 12,661,412 B2Granted
Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
AssigneeUniversity of Massachusetts
InventorsMiguel Sena Esteves, Ana Rita Batista, Sourav Roy Choudhury
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Abstract
The disclosure relates, in some aspects, to compositions and methods for enhanced delivery of a transgene to the central nervous system (CNS) of a subject. In some embodiments, the transgene is delivered by recombinant AAV (rAAV). In some embodiments, the method of enhancing transgene delivery comprises administering a blood brain barrier (BBB)-crossing molecule (e.g., K16ApoE) and an rAAV comprising a transgene to a subject.
Claims6
A method for delivering a transgene to CNS tissue in a subject, the method comprising co-administering to the subject:
- (i) an effective amount of a targeting composition, wherein the targeting composition comprises a hydrophilic peptide comprising the amino acid sequence of SEQ ID NO:
- 17;
- and,
- (ii) an effective amount of a recombinant adeno-associated virus (rAAV) comprising a nucleic acid comprising a promoter operably linked to a transgene and a capsid protein comprising an N-terminally grafted heterologous targeting peptide comprises or consists of a sequence selected from the group consisting of SEQ ID NOS:
- 15-17;
- wherein the co-administering comprises systemic co-injection.
3 more elements
Dependencies are inferred from the claim text.