US 12,285,497 B2
Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
Louise Rodino-Klapac, Columbus, OH (US); and Jerry R. Mendell, Columbus, OH (US)
Assigned to Research Institute at Nationwide Children's Hospital, Columbus, OH (US)
Filed by RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US)
Filed on Oct. 14, 2022, as Appl. No. 18/046,740.
Claims priority of provisional application 63/256,368, filed on Oct. 15, 2021.
Prior Publication US 2023/0139985 A1, May 4, 2023
Int. Cl. A61K 48/00 (2006.01); A61K 38/17 (2006.01); A61P 21/00 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0066 (2013.01) [A61K 38/1719 (2013.01); A61P 21/00 (2018.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01); C12N 2830/15 (2013.01)] 11 Claims
OG exemplary drawing
 
1. A polynucleotide sequence comprising a first nucleotide sequence at least 95% identical to the nucleotide sequence of SEQ ID NO: 1, wherein SEQ ID NO: 1 comprises a second nucleotide sequence encoding a β-sarcoglycan protein.