	US 12,258,573 B2
	Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
Louise Rodino-Klapac, Grove City, OH (US); Danielle Griffin, Canal Winchester, OH (US); and Jerry R. Mendell, Columbus, OH (US)
Assigned to Research Institute at Nationwide Children's Hospital, Columbus, OH (US)
Appl. No. 17/635,978
Filed by RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US)
PCT Filed Aug. 21, 2020, PCT No. PCT/US2020/047339 § 371(c)(1), (2) Date Feb. 16, 2022, PCT Pub. No. WO2021/035120, PCT Pub. Date Feb. 25, 2021.
Claims priority of provisional application 63/022,843, filed on May 11, 2020.
Claims priority of provisional application 63/014,934, filed on Apr. 24, 2020.
Claims priority of provisional application 62/889,749, filed on Aug. 21, 2019.
Prior Publication US 2022/0290180 A1, Sep. 15, 2022
Int. Cl. C12N 15/86 (2006.01); C07K 14/47 (2006.01); A61K 48/00 (2006.01); C07H 21/04 (2006.01)

CPC C12N 15/86 (2013.01) [C07K 14/4716 (2013.01); A61K 48/005 (2013.01); C07H 21/04 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01)]

13 Claims

1. A method of treating limb-girdle muscular dystrophy type 2D (LGMD2D) in a subject in need thereof comprising the step of intravenously administering a recombinant adeno-associated virus (rAAV), wherein the rAAV is administered using a systemic route of administration at a dose of about 5×10¹³vg/kg to about 2×10¹⁴vg/kg based on a supercoiled DNA or plasmid as the quantitation standard, and wherein the rAAV comprises a nucleotide sequence with at least 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, or 99% identity to SEQ ID NO: 4 and said nucleotide sequence encodes a protein that retains alpha-sarcoglycan activity, wherein the percentage identity is determined by aligning the sequence information with BLAST as the alignment tool.