Genome and transcriptome engineering with CRISPR/Cas as a precision medicine for Charcot-Marie-Tooth type 2L. University of Antwerp
Patients with Charcot-Marie-Tooth disease (CMT) have a hereditary motor and sensory neuropathy causing a length-dependent degeneration of their peripheral nerves. Most patients have a duplication on chromosome 17 resulting in a higher expression of the peripheral myelin protein 22 (PMP22). Finding ways to reduce PMP22 expression has led to the development of a treatment for this group of patients. However, a significant number of CMT patients ...