CMT4 (GDAP) Gene Therapy Research Program
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