Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.
Details
Title
ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHODS OF TREATING DYSTROPHIES
Inventors
McNally, Elizabeth : University of Chicago