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CONy 2021 | RNA- and DNA- based therapies for neurodegenerative & neuromuscular diseases

Aida Abu-Baker, PhD, Montreal Neurological Institute and Hospital, McGill University, Montreal, Canada, shares gene-based therapy approaches for treating neurodegenerative and neuromuscular diseases, focusing on the ongoing investigations which include RNA- and DNA-based therapies. Dr Abu-Baker discusses CRISPR/Cas9 and antisense oligonucleotide strategies for oculopharyngeal muscular dystrophy (OPMD) as well as an evolutionary algorithm for the design of RNA-activated hammerhead ribozymes that selectively cleave a mutant transcript. Additionally, promising gene therapy approaches for neurodegenerative diseases are discussed. This interview was conducted during the virtual 2021 World Congress on Controversies in Neurology (CONy).