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.

One of the major barriers for developing an AAV-based gene therapy for CMT is getting sufficient delivery of the therapeutic payload to the peripheral nerve, specifically the Schwann cells within the nerve for demyelinating CMTs. Current studies on AAV delivery systems studies in mice have shown that only a portion of Schwann cells in the peripheral nerve are transduced. This is more challenging when you want to target Schwann cells in large animals including NHPs and humans. This project aims to develop new AAV-based vehicles for delivering gene therapies more efficiently into the Schwann cells.
Dr. Afrooz Rashnonejad

Principal Investigator, Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital

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