Antisense suppression of the nonsense mediated decay factor Upf3b as a potential treatment for diseases caused by nonsense mutations.

Lulu HuangAudrey LowSagar S DamleMelissa M KeenanSteven KuntzSusan F MurrayBrett P MoniaShuling Guo

Published in: Genome biology (2018)

These data demonstrate that ASO-mediated reduction of the NMD factor Upf3b could be an effective and safe approach for the treatment of diseases caused by nonsense mutations.

Keyphrases

big data
artificial intelligence
climate change
replacement therapy