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ESGCT 2021 | AAV9-mediated gene therapy for CDKL5-deficiency disorder

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, describes ongoing research into the development of an adeno-associated virus (AAV) serotype 9-mediated gene replacement therapy to restore lost cyclin-dependent kinase-like 5 (CDKL5) protein expression seen in children with CDKL5 deficiency disorder (CDD). Administration through the cerebrospinal fluid (CSF) appears to be safer than system intravenous administration which requires higher dosages and therefore results in toxicity. He provides an overview of preclinical research in a CDD mouse model, as well as translational studies in non-human primates evaluating vector distribution and the gene expression profile of CDKL5 in the brain. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.