AES 2023 | Bench to bedside translation of gene therapy for rare epilepsies

Henry Lee, PhD, Boston Children’s Hospital, Harvard Medical School, Boston, MA, discusses some of the key challenges in bench to bedside translation of gene therapy for rare epilepsies. Achieving appropriate gene expression is one of the major challenges in gene replacement therapy. In his work in succinic semialdehyde dehydrogenase deficiency (SSADHD) gene therapy development, Dr Lee uses a native promoter so that the replacement gene mimics the endogenous gene’s expression pattern. Testing the efficacy of this approach is a key focus of his research team currently. Other regulatory mechanisms are also being studied, such as the use of microRNAs for self-regulation. Another consideration is the rate of expression and its relevance for therapeutic success. The impact of compensatory mechanisms resulting from pathway dysregulation needs to be considered when replacing genes. Finally, efficient virus delivery and precise targeting of specific brain regions is a key challenge when translating findings from animal models to humans. This interview took place during the American Epilepsy Society (AES) 2023 Annual Meeting congress in Orlando, FL.

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