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.
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA Presents New Genetic Landscape Analysis at The 2025 Annual Meeting of the Peripheral Nerve Society
The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth (CMT) disease research, presented a...
Accelerating Genetic Therapy for CMT4B1: A CMTA Research Update
With CMTA’s support, researchers at San Raffaele Scientific Institute in Milan, Italy, led by Alessandra...
AAV9-Mediated Gene Therapy to Treat Inherited CMT Neuropathies With Aberrant Myelin
This CMTA-funded study is testing a gene therapy approach for CMT4B1, a rare form of CMT that affects the myelin around nerves. Early results are encouraging, showing potential to restore nerve health by delivering a working copy of the affected gene.
Allele-Specific Silencing of MFN2 to Treat Peripheral Neuropathy
With CMTA support of $300,000, CMTA Strategy To Accelerate Research (CMTA-STAR) Advisory Board member Bruce...
