	US 12,104,163 B2
	Adeno-associated virus vectors for treatment of Rett syndrome
Colin O'Banion, Durham, NC (US)
Assigned to Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed by Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed on Aug. 19, 2021, as Appl. No. 17/406,723.
Claims priority of provisional application 63/067,668, filed on Aug. 19, 2020.
Prior Publication US 2022/0056478 A1, Feb. 24, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/86 (2006.01); A61K 38/18 (2006.01); A61K 48/00 (2006.01); A61P 25/28 (2006.01); C07K 14/475 (2006.01); C07K 14/61 (2006.01)

CPC C12N 15/86 (2013.01) [A61K 38/185 (2013.01); A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); A61P 25/28 (2018.01); C07K 14/475 (2013.01); C07K 14/61 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2830/008 (2013.01); C12N 2830/15 (2013.01); C12N 2830/38 (2013.01); C12N 2830/50 (2013.01)]

27 Claims

1. A nucleic acid comprising an adeno-associated virus (AAV) expression cassette, wherein the AAV expression cassette comprises, from 5′ to 3′:

a 5′ inverted terminal repeat (ITR);

a minimal human Arc gene promoter (hArcMin) set forth by SEQ ID no. 12;

a Rett Syndrome-associated gene; and

a 3′ ITR.