With CMTA support of $141,000, researchers led by Gabriel Corfas, PhD, at the Kresge Hearing...
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...
Modulating TEAD1 Activity in Charcot-Marie-Tooth Disease Type 1A
With CMTA support of $281,339, researchers at Albany Medical College, led by Sophie Belin, PhD, and Yannick Poitelon, PhD, are exploring the newly discovered TEAD1 pathway in Schwann cells that may regulate PMP22, the gene at the root of CMT1A.
ACT-CMT: CMT1A Natural History Study, Long-Term Progression, and Biomarkers
With $1.2M in CMTA funding, the ACT-CMT study is tracking long-term CMT1A progression and advancing biomarker development to prepare for future clinical trials.
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.
Targeting SREBP Regulation for CMT1A
With CMTA support of $74,000, researchers at the University of Wisconsin-Madison, led by CMTA Strategy...
Development of a Human-Derived Myelin-Containing Organoid as a Reference Model for CMT1A
With CMTA support, researchers at the University of Antwerp, led by Vincent Timmerman, PhD, are...
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...
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...
