Abstract
A Brief Note on Molecular Therapy
Molecular therapies for neuromuscular disorders are rapidly expanding and hold promise for devastating diseases with few options for treatment. Diseases targeted in this review include Duchenne, Becker, and the limb girdle muscular dystrophies. Strategies to repair defective genes involve exon skipping using antisense oligonucleotides or mutation suppression requiring small molecules that permit dose-dependent readthrough of stop codons. Clinical trials using these products have been completed or are underway. Gene replacement strategies targeting muscular dystrophies have been introduced into the clinic, and delivery potential is expanding by administration throughout the extremity using a vascular approach.
Author(s):
Raghavendra Tirupathi Govindaraju
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