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Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

30 Nov 2022

Abstract excerpt

Neurodegenerative disorders (NDDs), such as Alzheimer's disease (AD) and Parkinson's Disease (PD), are a group of heterogeneous diseases that mainly affect central nervous system (CNS) functions. A subset of NDDs exhibit CNS dysfunction and muscle degeneration, as observed in Gangliosidosis 1 (GM1) and late stages of PD. Neuromuscular disorders (NMDs) are a group of diseases in which patients show primary...

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PMID: 36556200
DOI: 10.3390/jpm12121979

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