Gene Therapy Approach to Treating MECP2 Duplication Syndrome

Kevin Foust, PhD | Ohio State University

$40,000 AWARDED

The duplication syndrome is caused by having an extra copy (or two) of the MECP2 gene and sometimes other genes in the vicinity. In theory, reducing the amount of MeCP2 protein should improve the disease. Dr. Foust will use adeno-associated virus (AAV) to deliver RNA interference to lower the amount of MeCP2 protein. If successful the project will provide proof-of-concept data showing that MeCP2 reduction is a therapeutic option for patients.

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