CMT4 Gene Therapy Research Program

Blue glowing neural network nodes connected by luminescent pathways against dark background.

About CMT4A / GDAP1

CMT4 is a rare subtype of CMT, a genetic, neurological disorder that causes damage to the peripheral nerves, tracts of nerve cell fibers that connect the brain and spinal cord to muscles and sensory organs.

CMT4 is a subtype of CMT that is inherited in an autosomal recessive pattern.

“As genetic therapies quickly become a real option for CMT patients, this new HNF project is very exciting for patients, physicians, and researchers alike”

, Dr. Stephen Züchner, MD, PhD Professor for Human Genetics and Neurology Chair, Dr. John T. Macdonald Foundation Department of Human Genetics Co-Director, John P. Hussman Institute for Human Genomics University of Miami Miller School of Medicine and founder of The Genesis Project

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