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ESGCT 2021 | Challenges with translating gene therapies for neurodevelopmental disorders

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, discusses the major challenges associated with the translation of gene therapies for neurodevelopmental disorders (NDDs) to the clinic. He explains the difficulty in translating therapies for NDDs from mouse models into non-human primate models and humans, given the increased brain size and complexity which support higher-level brain function. In addition, when attempting to use gene therapy approaches to restore lost gene expression seen in NDDs such as CDKL5-deficiency disorder (CDD), it can be difficult to model and predict which neurons need to be targeted in order to achieve lasting benefit in patients. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.