	US 12,454,702 B2
	Minigene therapy
Guangping Gao, Worcester, MA (US); and Hemant Khanna, Worcester, MA (US)
Assigned to University of Massachusetts, Westborough, MA (US)
Filed by University of Massachusetts, Westborough, MA (US)
Filed on Jun. 30, 2023, as Appl. No. 18/345,366.
Application 18/345,366 is a continuation of application No. 16/500,238, granted, now 11,739,346, previously published as PCT/US2018/026230, filed on Apr. 5, 2018.
Claims priority of provisional application 62/481,727, filed on Apr. 5, 2017.
Prior Publication US 2024/0076692 A1, Mar. 7, 2024
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/63 (2006.01); A61K 9/00 (2006.01); A61K 35/761 (2015.01); A61P 27/02 (2006.01); C07H 21/04 (2006.01); C07K 14/47 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01); A01K 67/00 (2006.01); A61K 48/00 (2006.01)

CPC C12N 15/86 (2013.01) [A61K 9/0048 (2013.01); A61K 35/761 (2013.01); A61P 27/02 (2018.01); C07K 14/47 (2013.01); C12N 7/00 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01)]

8 Claims

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

(i) arAAV vector encoding a CEP290 protein fragment comprising the amino acid sequence set forth in SEQ ID NO: 3, wherein the CEP290 protein fragment comprises no more than 700 contiguous amino acids of SEQ ID NO: 1, and wherein the CEP290 protein fragment lacks amino acids 1695 to 1966 of SEQ ID NO: 1, and

(ii) one or more AAV capsid proteins.