Abstract:Background: Gene therapy has proved to be a boon for neuromuscular diseases. A concept introduced in the early 1960’s, has been put into clinical practice in the past 5-10 years. The process by which a healthy gene replaces a defective gene in a human body through vectors is truly pathbreaking. Patients with inherited degenerative neuromuscular disorders such as spinal muscular atrophy, and muscular dystrophies undergo progressive deterioration and eventually death. Gene therapy and other adjunctive clinical a… Show more
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