Dual base editor catalyzes both cytosine and adenine base conversions in human cells.
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Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease.
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Silencing hepatic MCJ attenuates non-alcoholic fatty liver disease (NAFLD) by increasing mitochondrial fatty acid oxidation.
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mRNA structural dynamics shape Argonaute-target interactions.
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G4C2 Repeat RNA Initiates a POM121-Mediated Reduction in Specific Nucleoporins in C9orf72 ALS/FTD
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Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease
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