	US 12,391,928 B2
	Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
Zarife Sahenk, Columbus, OH (US)
Assigned to Research Institute at Nationwide Children's Hospital, Columbus, OH (US)
Appl. No. 17/255,488
Filed by RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US)
PCT Filed Jun. 28, 2019, PCT No. PCT/US2019/039893 § 371(c)(1), (2) Date Dec. 23, 2020, PCT Pub. No. WO2020/006458, PCT Pub. Date Jan. 2, 2020.
Claims priority of provisional application 62/865,081, filed on Jun. 21, 2019.
Claims priority of provisional application 62/691,934, filed on Jun. 29, 2018.
Prior Publication US 2021/0277362 A1, Sep. 9, 2021
Int. Cl. C12N 7/00 (2006.01); A61K 48/00 (2006.01); A61P 21/00 (2006.01); C12N 9/64 (2006.01); C12N 15/86 (2006.01)

CPC C12N 7/00 (2013.01) [A61K 48/0058 (2013.01); A61P 21/00 (2018.01); C12N 9/6472 (2013.01); C12N 15/86 (2013.01); C12N 2750/14131 (2013.01); C12N 2750/14143 (2013.01)]

4 Claims

1. A recombinant adeno-associated virus (rAAV) comprising a polynucleotide which comprises nucleotides 1 to 3977 of SEQ ID NO: 1, wherein the rAAV comprises an AAV-8, an AAV-9, or an AAV rh.74 capsid protein, or a variant of each thereof.