TREATMENT OF MUSCULAR DYSTROPHY USING PEPTIDE NUCLEIC ACID ( PNA)

摘要

A method of correcting expression of a gene in a human cell having a muscle disease or muscular dystrophy phenotype, wherein without correction the gene fails to express functional protein due to one or more mutations, said method comprising delivering to the cell a peptide nucleic acid (PNA) comprising a sequence capable of targeting a sequence responsible for exon skipping in the mutated pre-mRNA at an exon to be skipped or included, wherein expression is corrected by the PNA inducing exon skipping or inclusion and thereby correcting the expression of said mutated pre-mRNA.