@article{PATENT,
      recid = {9449},
      title = {COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE  MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING},
      number = {PATENT},
      month = {Apr},
      year = {2022},
      abstract = {The 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 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.},
      url = {http://knowledge.uchicago.edu/record/9449},
}