tRNA Gene Replacement Therapy for tRNA-Synthetase Related Charcot-Marie-Tooth Disorders

Charcot-Marie-Tooth Disease (CMT), a group of inherited disorders causing peripheral nerve damage, affects 1 in 3,300 people worldwide. Individuals experience weakness in their muscles, sensation loss and muscle contractions in their feet, legs, arms and hands. Recent research shows overexpression of tRNA-glycine can ease peripheral neuropathy caused by Glycyl-tRNA synthetase (GARS) mutations. Principal investigator Scott Harper of Nationwide Children’s Hospital’s Center for Gene Therapy developed a gene therapy to overexpress relevant tRNAs associated with various tRNA synthetase-related disorders like CMT. This therapy would improve an individual’s quality of life by lessening the pain, weakness, and numbness from the nerve damage.

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