Therapeutic Targeting of RNA Splicing in Cancer

RNA splicing is a key regulatory step in the proper control of gene expression. It is a highly dynamic process orchestrated by the spliceosome, a macro-molecular machinery that consists of protein and RNA components. The dysregulation of RNA splicing has been observed in many human pathologies rangi...

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Main Authors: Elizabeth A. Bonner, Stanley C. Lee
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:Genes
Subjects:
Online Access:https://www.mdpi.com/2073-4425/14/7/1378
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author Elizabeth A. Bonner
Stanley C. Lee
author_facet Elizabeth A. Bonner
Stanley C. Lee
author_sort Elizabeth A. Bonner
collection DOAJ
description RNA splicing is a key regulatory step in the proper control of gene expression. It is a highly dynamic process orchestrated by the spliceosome, a macro-molecular machinery that consists of protein and RNA components. The dysregulation of RNA splicing has been observed in many human pathologies ranging from neurodegenerative diseases to cancer. The recent identification of recurrent mutations in the core components of the spliceosome in hematologic malignancies has advanced our knowledge of how splicing alterations contribute to disease pathogenesis. This review article will discuss our current understanding of how aberrant RNA splicing regulation drives tumor initiation and progression. We will also review current therapeutic modalities and highlight emerging technologies designed to target RNA splicing for cancer treatment.
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spelling doaj.art-7e2d84932c9c4f4f8b709999c4682ec02023-11-18T19:29:33ZengMDPI AGGenes2073-44252023-06-01147137810.3390/genes14071378Therapeutic Targeting of RNA Splicing in CancerElizabeth A. Bonner0Stanley C. Lee1Molecular and Cellular Biology Graduate Program, University of Washington, Seattle, WA 98195, USATranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA 98109, USARNA splicing is a key regulatory step in the proper control of gene expression. It is a highly dynamic process orchestrated by the spliceosome, a macro-molecular machinery that consists of protein and RNA components. The dysregulation of RNA splicing has been observed in many human pathologies ranging from neurodegenerative diseases to cancer. The recent identification of recurrent mutations in the core components of the spliceosome in hematologic malignancies has advanced our knowledge of how splicing alterations contribute to disease pathogenesis. This review article will discuss our current understanding of how aberrant RNA splicing regulation drives tumor initiation and progression. We will also review current therapeutic modalities and highlight emerging technologies designed to target RNA splicing for cancer treatment.https://www.mdpi.com/2073-4425/14/7/1378alternative splicingsplicing factor mutationsspliceosome inhibitorsanti-sense oligonucleotidesimmuno-oncology therapies
spellingShingle Elizabeth A. Bonner
Stanley C. Lee
Therapeutic Targeting of RNA Splicing in Cancer
Genes
alternative splicing
splicing factor mutations
spliceosome inhibitors
anti-sense oligonucleotides
immuno-oncology therapies
title Therapeutic Targeting of RNA Splicing in Cancer
title_full Therapeutic Targeting of RNA Splicing in Cancer
title_fullStr Therapeutic Targeting of RNA Splicing in Cancer
title_full_unstemmed Therapeutic Targeting of RNA Splicing in Cancer
title_short Therapeutic Targeting of RNA Splicing in Cancer
title_sort therapeutic targeting of rna splicing in cancer
topic alternative splicing
splicing factor mutations
spliceosome inhibitors
anti-sense oligonucleotides
immuno-oncology therapies
url https://www.mdpi.com/2073-4425/14/7/1378
work_keys_str_mv AT elizabethabonner therapeutictargetingofrnasplicingincancer
AT stanleyclee therapeutictargetingofrnasplicingincancer