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1. Introduction Neurodegenerative diseases, such as Alzheimer’s disease (AD), Parkinson’s disease (PD), Huntington’s disease (HD) and Amyotrophic Lateral Sclerosis (ALS) are characterized by a progressive loss of specific neuronal cell populations, in addition to other features such as abnormal protein aggregation, mitochondrial dysfunction, pro-oxidative and pro-inflammatory mechanisms [1,2,3].
Introduction Gene therapies provide several major advantages compared to all other therapeutics which make them uniquely valuable for the treatment of neurodegenerative diseases (NDDs). First, gene therapies must be delivered intracellularly in order to function (Ingusci et al., 2019). Though this poses a challenge, once successfully delivered, gene therapies are able to directly modify protein production at the source (Ingusci et al., 2019).
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