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CONy 2021 | Challenges in developing gene-based therapies for neurodegenerative & neuromuscular diseases

Aida Abu-Baker, PhD, Montreal Neurological Institute and Hospital, McGill University, Montreal, Canada, discusses the challenges encountered during the development of gene-based therapies for neurodegenerative and neuromuscular diseases. Firstly, disease pathophysiology remains incompletely characterized for most disorders, and animal models are not as advanced as in other fields. Additionally, there are no easily measured biomarkers for the rate of progression. Finally, brain drug delivery remains a significant hurdle in the treatment of neurodegenerative diseases. Dr Abu-Baker describes drug delivery strategies for gene-based therapies that have been successful and others that are currently under development. This interview was conducted during the virtual 2021 World Congress on Controversies in Neurology (CONy).