Gene therapy 2.0: genetic therapies for non-genetic disorders in neurology

“...the potential benefits from harnessing genetic therapies for relief in a widening range of non-genetic disorders is worth a deliberate effort...”

Gene therapy has largely been confined to replacing faulty genes in rare genetic disorders, but a growing body of evidence suggests genetic tools can also be used to correct abnormal neuronal activity in diseases with no genetic cause. Advances in neuroscience have yielded a broad toolkit including miRNAs, ASOs, and CRISPRa capable of precisely modulating specific circuits, with early clinical successes already demonstrated in Parkinson’s disease and epilepsy. Key challenges remain, including the invasiveness of direct brain delivery and the permanence of virally-delivered modifications, though reversible approaches such as chemogenetics and inducible systems offer a path forward.

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