Technologies

Gene therapy to treat Charcot-Marie-Tooth (CMT) neuropathies.

Charcot-Marie-Tooth (CMT) neuropathies are one of the most common inherited neurological conditions affecting 1 in 2,500 people in the United States. Both children and adults are affected, causing sensory and motor dysfunction, pain, and a need for ambulatory aids. Researchers at Nationwide Children’s Hospital have developed a gene therapy approach that delivers neurotrophic factor NT-3 by intramuscular injection to promote nerve regeneration in CMT disease as well as other nerve diseases with impaired nerve regeneration.