To Splice or Not to Splice, That Is the Treatment

Mariano A. Garcia-Blanco

doi:10.1016/j.chembiol.2020.12.002

To Splice or Not to Splice, That Is the Treatment

Mariano A. Garcia-Blanco

Research output: Contribution to journal › Comment/debate › peer-review

Abstract

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 language	English (US)
Pages (from-to)	1453-1455
Number of pages	3
Journal	Cell Chemical Biology
Volume	27
Issue number	12
DOIs	https://doi.org/10.1016/j.chembiol.2020.12.002
State	Published - Dec 17 2020
Externally published	Yes

ASJC Scopus subject areas

Biochemistry
Molecular Medicine
Molecular Biology
Pharmacology
Drug Discovery
Clinical Biochemistry

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10.1016/j.chembiol.2020.12.002

Cite this

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title = "To Splice or Not to Splice, That Is the Treatment",

abstract = "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.",

author = "Garcia-Blanco, {Mariano A.}",

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year = "2020",

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doi = "10.1016/j.chembiol.2020.12.002",

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DO - 10.1016/j.chembiol.2020.12.002

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To Splice or Not to Splice, That Is the Treatment

Abstract

ASJC Scopus subject areas

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