Splice-switching as cancer therapy

Amina Jbara, Zahava Siegfried, Rotem Karni*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations


In light of recent advances in RNA splicing modulation as therapy for specific genetic diseases, there is great optimism that this approach can be applied to treatment of cancer as well. Dysregulation of alternative RNA splicing is a common aberration detected in many cancers and thus, provides an attractive target for therapeutics. Here, we present and compare two promising approaches that are currently being investigated to manipulate alternative splicing and their potential use in therapy. The first strategy makes use of splice-switching oligonucleotides, whereas the second strategy uses CRISPR (clustered regularly interspaced short palindromic repeat Cas (CRISPR-associated) technology. We will discuss both the challenges and limitations of these technologies and progress being made to implement splice-switching as a potential cancer therapy.

Original languageAmerican English
Pages (from-to)140-148
Number of pages9
JournalCurrent Opinion in Pharmacology
StatePublished - Aug 2021

Bibliographical note

Funding Information:
The authors thank Ela Elyada for critically reading the manuscript. Work in the Karni laboratory is supported by grants from the Israel Science Foundation (ISF Grant 1510/17 ), the Len and Susan Mark Initiative for Ovarian and Uterine/MMMT Cancers , ICRF , and the Alex U. Soyka Pancreatic Cancer Research grant ( CFHU ). Figures were created with http://BioRender.com .

Publisher Copyright:
© 2021 Elsevier Ltd


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