GRIN2D RNAi Gene Therapy to Treat Epilepsy

Overexpression of the GRIN2D gene can cause Developmental and Epileptic Encephalopathy (DEE) in which individuals may experience developmental delays or intellectual disabilities, epilepsy, abnormal muscle tone, movement disorders, autism spectrum disorder and cortical visual impairment. Currently, only supportive care is available. Genetic Researchers at Nationwide Children’s Hospital developed a RNAi gene therapy to treat epilepsy caused by GRIN2D. They propose decreasing the expression of GRIN2D through a gene-level specific reagent which will knock down the mRNA containing the variant postnatally. As a result, reducing the possibility of children developing GRIN2D-related DEE.

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