Listando por materia "Gene therapy"
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Malin restoration as proof of concept for gene therapy for Lafora disease
(Brain Communications 2022, 4(4), fcac168, 2022)Lafora disease is a fatal neurodegenerative childhood dementia caused by loss-of-function mutations in either the laforin or malin gene. The hallmark of the disease is the accumulation of abnormal ... -
Polymer-based coating of adeno-associated viral particles as a new strategy to evade immune response for DMD treatment
(Journal of Controlled Release 2025, 384, 2025-08-10)Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene, leading to progressive muscle degeneration. Gene therapy using adeno-associated virus (AAV) ...