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MECP2 Duplication Syndrome HuidaGene Therapeutics has developed a therapy for MECP2 duplication syndrome (MDS) which uses their RNA editing platform to reduce the amount of MECP2 RNA and ultimately the amount of MECP2 protein
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Cell Mol Life Sci , 2014, 71(4): 683-697
Specifically, SIRT3 deacetylates and inhibits aldehyde dehydrogenase 2, which prevents the conversion of acetaldehyde to acetate, potentially leading to acetaldehyde toxicity 183
