Dr. Afrooz Rashnonejad, Nationwide Children’s Hospital, Columbus, Ohio
Gene Therapy for Several Types of CMT
The development of safe and effective gene therapies for CMT requires the delivery of the therapeutic genetic payload into the peripheral nervous system, specifically targeting Schwann cells for certain CMT types, including but not limited to CMT1A, 1B, 4C, 4J, and 1X. Special carriers called adeno-associated viral (AAV) vectors currently serve as the major delivery method for gene therapies. Dr. Afrooz Rashnonejad, PhD, a principal investigator in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s will work to develop new AAV-based (adeno-associated viral) vehicles for delivering gene therapies more efficiently into the Schwann cells. This will improve treatment efficacy and safety of gene therapies for several types of CMT, including the most prevalent form, CMT1A.
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