CMTA-supported scientists are using CRISPR to go after the genetic root of CMT1A and CMT1B. Early results are changing what’s possible. See how this research is shifting from bold ideas to future realities.
Targeting the Cell’s Clean-Up System in CMT1
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.
CMTA’s $450K Investment Targets New Approach to Treatment for Patients with CMT1A and CMT1B
CMTA announced a $450,000 investment supporting research into a cGMP-based therapeutic approach for CMT1A and CMT1B, building on prior CMTA-funded studies.
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...
CMTA Funds Preclinical Work on Modulating Unfolded Protein Response in CMT1B Mice
CMTA has funded a preclinical study exploring whether modulating the unfolded protein response (UPR) can address disease mechanisms in CMT1B. The project will evaluate activation of the ATF6 pathway in CMT1B mouse models to determine whether this approach can reduce cellular stress linked to myelin dysfunction and inform future therapeutic development.
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,...
