CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments.
CMTA-STAR Delivery Science: Four Approaches for CMT
CMTA is funding four research projects targeting a fundamental barrier to CMT treatment: getting therapies past the biological defenses protecting peripheral nerves. The $523,000 CMTA-STAR investment supports complementary delivery approaches, including gene editing, RNA medicine, and nanoparticles, across multiple CMT subtypes.
Peptide-LNPs Deliver siRNA Against PMP22 in CMT1A
With combined support totaling $102,968 from CMTA, Shark Tooth Bio, and the National Research Council...
Blood–Brain Barrier-Crossing Conjugates for Treating CMT
CMTA-funded research at Mount Sinai is studying how RNA medicines can be delivered into nerve cells in CMT, overcoming biological barriers that block treatment.
STEP Platform for Delivering Genome Editing Therapy in CMT
CMTA-funded Yale researchers are testing STEP, a non-viral delivery system for genome-editing medicines in CMT.
Can We Fix the Code? A Look at Genetic Therapies for CMT
Can we fix the genes that cause CMT? CMTA’s Dr. Katherine Forsey breaks down the science behind genetic therapies and what’s heading toward clinical trials.
Nanoparticle Gene Delivery to Schwann Cells for CMT
With joint funding of $299,992 from CMTA and the Muscular Dystrophy Association (MDA), researchers at...
