COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

摘要

Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

主权项

1. A method of treating Duchenne muscular dystrophy or Becker muscular dystrophy in a human subject comprising administering about 30 mg/kg to about 50 mg/kg of a composition comprising an antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides complementary to a target region in an exon of the human dystrophin gene, wherein the antisense oligonucleotide specifically hybridizes to the target region inducing exon skipping, thereby treating Duchenne muscular dystrophy or Becker muscular dystrophy in the subject.