To Splice or Not to Splice, That Is the Treatment.

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Garcia-Blanco, MA

Published Date

  • December 17, 2020

Published In

Volume / Issue

  • 27 / 12

Start / End Page

  • 1453 - 1455

PubMed ID

  • 33338466

Electronic International Standard Serial Number (EISSN)

  • 2451-9448

Digital Object Identifier (DOI)

  • 10.1016/j.chembiol.2020.12.002


  • eng

Conference Location

  • United States