	US 12,031,147 B2
	Adeno-associated virus virions with variant capsids and methods of use thereof
John G. Flannery, Berkeley, CA (US); Scott F. Geller, El Cerrito, CA (US); and Karen I. Guerin, Braintree, MA (US)
Assigned to The Regents of the University of California, Oakland, CA (US); and Novartis AG, Basel (CH)
Filed by The Regents of the University of California, Oakland, CA (US); and Novartis AG, Basel (CH)
Filed on May 27, 2021, as Appl. No. 17/332,855.
Claims priority of provisional application 63/187,154, filed on May 11, 2021.
Claims priority of provisional application 63/032,206, filed on May 29, 2020.
Prior Publication US 2021/0371879 A1, Dec. 2, 2021
Int. Cl. C12N 15/86 (2006.01); C07K 14/005 (2006.01)

CPC C12N 15/86 (2013.01) [C07K 14/005 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14143 (2013.01)]

24 Claims

1. A recombinant adeno-associated virus (rAAV) virion comprising:

a variant AAV2 capsid protein VP1, wherein the variant AAV2 capsid protein comprises an insertion of a heterologous peptide comprising the amino acid sequence set forth in any one of SEQ ID NOs:2 and 3 between amino acids 570 and 611 of the AAV2 capsid protein VP1, and wherein the variant AAV2 capsid protein confers increased infectivity of a retinal cell compared to the infectivity of the retinal cell by a control AAV virion comprising a corresponding parental AAV capsid protein not comprising the heterologous peptide; and

a heterologous nucleic acid comprising one or more nucleotide sequences encoding one or more heterologous gene products.