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Long-term effect of gene therapy on Lebers congenital amaurosis

Another deleterious mechanism associated with mitochondrial dysfunction is the limitation in ATP production that can cause an energy crisis in neurons (Nicholls, 2013) and calpain-activation are linked to AD (Saito et al., Not only are mitochondria associated with UPS function in regards to ATP production and the generation of ROS, the UPS is also linked to mitochondrial function, as recent studies revealed that the UPS degrades mitochondrial proteins and thus contributes to mitochondrial quality control (Taylor and Rutter, 2011)

The NNMT-blocking mechanism of 5-amino-1MQ has been shown to increase levels of the cofactor NAD+, whose replenishment in organisms promotes muscular, cognitive, and immune function, as well as increased lifespan [4, 5]

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