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Investigating the use of oligonucleotides for the treatment of muscular dystrophy
Antisense oligonucleotide therapy is one of the most promising strategies for treatment of myotonic dystrophy type 1 (DM1), type 2 (DM2) and Duchene muscular dystrophy (DMD). These three diseases have nuclear retained mRNA, suitable for antisense therapy targeting. The delivery of oligonucleotides
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