In CMT1A and CMT1B, the cell's protein clean-up system breaks down, allowing harmful proteins to build up and damage peripheral nerves. CMTA-funded researcher Jordan VerPlank, PhD, is working to reactivate that system using medicines already tested in people.
Gene Editing Strategies for Demyelinating CMT
CMTA-supported researchers are advancing CRISPR-based gene editing for CMT1A and CMT1B, laying the foundation for mutation-specific therapies.
Nanoparticle-Based Gene Delivery to Schwann Cells for Treating CMT Disease
With joint funding of $299,992 from CMTA and the Muscular Dystrophy Association (MDA), researchers at...
Testing New UPR-Targeted Therapies for CMT1B
Researchers at the Ospedale San Raffaele (OSR) Scientific Institute in Milan, Italy, supported by the...
Schwann Cell-Targeted Gene Therapy Approaches to Treat CMT1A and Other Demyelinating Neuropathies
With CMTA support of over $160,000, an international team of researchers is developing genetic therapy...
1B Biomarkers and Outcome Measures
With CMTA support of $529,971, an international team of researchers from the Inherited Neuropathy Consortium,...
