Nicotinamide riboside, a vitamin B3, is effective to delayed early- and late-stage disease progression, via mitochondrial biogenesis in skeletal muscle and brown adipose tissue, thereby preventing mitochondrial ultrastructure abnormalities and mtDNA deletion formation. NR further stimulated mitochondrial unfolded protein response, suggesting its protective role in mitochondrial disease.
Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
Genome-editing reduces cholesterol [mouse]
Khan NA, Auranen M, Paetau I, Pirinen E, Euro L, Forsstrom S, Pasila L, Velagapudi V, Carroll CJ, Auwerx J, Suomalainen A. EMBO Mol Med. 2014 Apr 6;6(6):721-31.
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