To Splice or Not to Splice, That Is the Treatment

Mariano A. Garcia-Blanco

Research output: Contribution to journalComment/debatepeer-review


In this issue of Cell Chemical Biology, Shibata et al. (2020) rescue expression of CFTR from a defective gene by inhibiting splicing factors required for the inclusion of a pathogenic pseudo exon. Their work highlights the untapped potential of RNA splicing as a therapeutic target.

Original languageEnglish (US)
Pages (from-to)1453-1455
Number of pages3
JournalCell Chemical Biology
Issue number12
StatePublished - Dec 17 2020
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Molecular Biology
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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