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.
Schwann Cell-Targeted Peptide-LNPs for Delivery of 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
With CMTA support of $54,051, researchers led by Yizhou Dong, PhD, at the Icahn School...
Preliminary Characterization of STEP Platform for Delivery of Genome Editing Therapy for CMT
With CMTA support of $66,000, researchers led by Jiangbing Zhou, PhD, at Yale University are...
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-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...
