Method and material for activating internal ribosome entry site in exon 5 of DMD gene

摘要

Problem to be solved: to provide a method and material for activating an internal ribosome entry site within the exon 5 of the DMD gene.The present invention relates to a delivery of an oligomer for treating a patient having 5 'mutations in a DMD gene except for DMD exon 2 duplication.The present invention provides methods and materials for activating the internal ribosome entry site within exon 5 of the DMD gene and causing translation of the functional cleavage isoforms of dystrophin.The method and material may be used for the treatment of muscular dystrophy resulting from 5 'mutations in the DMD gene such as Duchenne muscular dystrophy or Becker muscular dystrophy.No selection