Susan Ruediger on Partnering to Develop CMT Gene Therapies

“Dr. Afrooz Rashnonejad at Nationwide Children's Hospital has found an approach that will address all of the mutations in myelin protein zero (MPZ). And she hopes that she can knock down and replace the way that that gene expresses in a way that addresses all people and all mutations of CMT1B.”

The CMT Research Foundation (CMTRF) is working to help researchers find treatments for Charcot-Marie-Tooth disease (CMT) by funding promising projects, including cell and gene therapy approaches, to address unmet needs in CMT.

The CMTRF recently signed an agreement with Nationwide Children’s Hospital and awarded a $500,000 grant to Afrooz Rashnonejad, PhD, principal investigator at Nationwide and Assistant Professor at the Ohio State University’s Department of Pediatrics in Columbus, Ohio. Rashnonejad is investigating a knock down and replace approach to silence the mutated MPZ gene and replace it with a functioning gene.

CGTLive spoke with Susan Ruediger, founder and chief mission officer, CMT Research Foundation, to learn more about the new grant and research that it is funding. She discussed the potential of the gene therapy research approach. She also discussed how such partnerships benefit the researcher, the CMTRF, and patients.

REFERENCE

CMT Research Foundation Awards Grant to Nationwide Children’s Hospital’s Dr. Afrooz Rashnonejad to Develop Gene Therapy to Cure or Alleviate CMT1B. News release CMTRF. July 18, 2022. https://cmtrf.org/nationwide-childrens-hospital-cmt1b-press-release/