Scientists Unveil Promising New Treatment for a Common Hereditary Nerve Disease
Researchers at Tokyo Medical and Dental University have developed a groundbreaking genome-editing technique to treat Charcot–Marie–Tooth disease by reducing PMP22 protein levels, showing potential for a new clinical therapy in a field with limited existing treatments. AAV gene therapy-based genome editing recovered myelination in human CMT1A patient nerve differentiated from iPS cells. Credit: Department of Neuropathology, TMDUScientists at Tokyo Medical and Dental University have created a new genome-editing method that…