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...
CMTA’s $300K Investment in CRISPR for CMT2A
The Charcot-Marie-Tooth Association (CMTA) is investing $300,000 in a groundbreaking gene editing research project at...
CMTA’s $300K Investment in CRISPR for CMT2A
CMTA announced a $300,000 CMTA-STAR investment supporting CRISPR-based gene editing research aimed at selectively silencing disease-causing MFN2 mutations in CMT2A.
Encouraging News in CMT Research from CMTA-STAR Alliance Partner Alesta Therapeutics
Alesta Therapeutics, a CMTA-STAR Alliance Partner focused on developing therapies for rare diseases, recently announced...
Gene Replacement Therapy for CMT4C IND-Enabling Studies
CMTA and families are driving Project Foresee to bring genetic therapy for CMT4C into clinical trials. Learn how this effort is moving toward patients.
Gene Replacement Therapy for CMT4A Neuropathy in Rodents
With CMTA support of $248,950, researchers at the University of Texas Southwestern, led by CMTA’s...
CMTA’s $240K Investment Targets Gene Replacement Therapy in CMT4 Subtypes
CMTA announced a $240,000 investment AAV-based gene replacement for CMT4A, with potential implications for CMT4B1 and CMT4D.
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.
ASO Approaches for CMT2E
With CMTA support of $225,483, researchers at the University of Miami, led by CMTA Strategy...
