000007393 001__ 7393
000007393 005__ 20240523050954.0
000007393 024__ $$aUS 10801029 B2
000007393 024__ $$dUS 201916395741 A
000007393 037__ $$aPATENT
000007393 041__ $$aEnglish
000007393 245__ $$aCompositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
000007393 260__ $$c2020-10-13
000007393 260__ $$g2019-04-26
000007393 336__ $$aPatent
000007393 520__ $$aThe invention is directed to one or more antisense polynucleotides and their use in pharmaceutical compositions in a strategy to induce exon skipping in the γ-sarcoglycan gene in patients suffering from Limb-Girdle Muscular Dystrophy-2C (LGM-D2C) or in patients at risk of such a disease. The invention also provides methods of preventing or treating muscular dystrophy. e.g., LGMD2C, by exon skipping in the gamma sarcoglycan gene using antisense polynucleotides. Accordingly, in some aspects the invention provides an isolated antisense oligonucleotide, wherein the oligonucleotide specifically hybridizes to an exon target region of a γ-sarcoglycan RNA. In another aspect, the invention provides a method of inducing exon-skipping of a gamma sarcoglycan RNA, comprising delivering an antisense oligonucleotide or a composition to a cell.
000007393 690__ $$aBiological Sciences Division
000007393 691__ $$aMedicine
000007393 7011_ $$aMcNally, Elizabeth$$uUniversity of Chicago
000007393 7011_ $$aWyatt, Eugene$$uUniversity of Chicago
000007393 7121_ $$aUniversity of Chicago
000007393 7121_ $$aNorthwestern University
000007393 7131_ $$aUniversity of Chicago
000007393 7131_ $$aNorthwestern University
000007393 8564_ $$9879e15ab-49f2-4400-beef-f1b99fd4781a$$s2170437$$uhttps://knowledge.uchicago.edu/record/7393/files/US10801029.pdf$$ePublic
000007393 909CO $$ooai:uchicago.tind.io:7393$$pGLOBAL_SET
000007393 983__ $$aPatent