	US 11,739,346 B2
	Minigene therapy
Guangping Gao, Westborough, MA (US); and Hemant Khanna, Sutton, MA (US)
Assigned to University of Massachusetts, Boston, MA (US)
Appl. No. 16/500,238
Filed by University of Massachusetts, Boston, MA (US)
PCT Filed Apr. 5, 2018, PCT No. PCT/US2018/026230 § 371(c)(1), (2) Date Oct. 2, 2019, PCT Pub. No. WO2018/187552, PCT Pub. Date Oct. 11, 2018.
Claims priority of provisional application 62/481,727, filed on Apr. 5, 2017.
Prior Publication US 2020/0056204 A1, Feb. 20, 2020
Int. Cl. C07H 21/04 (2006.01); C12N 15/63 (2006.01); A61K 31/70 (2006.01); C12N 15/86 (2006.01); A61P 27/02 (2006.01); A61K 9/00 (2006.01); A61K 35/761 (2015.01); C07K 14/47 (2006.01); C12N 7/00 (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)]

16 Claims

1. An isolated nucleic acid comprising:

(i) a first region comprising a first adeno-associated virus (AAV) inverted terminal repeat (ITR), or a variant thereof; and,

(ii) a second region comprising a transgene encoding a CEP290 protein fragment, wherein the CEP290 protein fragment comprises at least 500 contiguous amino acids of SEQ ID NO: 1, wherein the CEP290 fragment comprises no more than 700 contiguous amino acids of SEQ ID NO: 1, wherein the CEP290 fragment comprises or consists of the sequence set forth in SEQ ID NO: 3, and wherein the CEP290 fragment does not comprise amino acids 1695 to 1966 of SEQ ID NO: 1.