Small molecule inhibition of RNA binding proteins in haematologic cancer

In recent years, advances in biomedicine have revealed an important role for post-transcriptional mechanisms of gene expression regulation in pathologic conditions. In cancer in general and leukaemia specifically, RNA binding proteins have emerged as important regulator of RNA homoeostasis that are...

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Main Authors: Amit K. Jaiswal, Michelle L. Thaxton, Georgia M. Scherer, Jacob P. Sorrentino, Neil K. Garg, Dinesh S. Rao
Format: Article
Language:English
Published: Taylor & Francis Group 2024-12-01
Series:RNA Biology
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Online Access:https://www.tandfonline.com/doi/10.1080/15476286.2024.2303558
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author Amit K. Jaiswal
Michelle L. Thaxton
Georgia M. Scherer
Jacob P. Sorrentino
Neil K. Garg
Dinesh S. Rao
author_facet Amit K. Jaiswal
Michelle L. Thaxton
Georgia M. Scherer
Jacob P. Sorrentino
Neil K. Garg
Dinesh S. Rao
author_sort Amit K. Jaiswal
collection DOAJ
description In recent years, advances in biomedicine have revealed an important role for post-transcriptional mechanisms of gene expression regulation in pathologic conditions. In cancer in general and leukaemia specifically, RNA binding proteins have emerged as important regulator of RNA homoeostasis that are often dysregulated in the disease state. Having established the importance of these pathogenetic mechanisms, there have been a number of efforts to target RNA binding proteins using oligonucleotide-based strategies, as well as with small organic molecules. The field is at an exciting inflection point with the convergence of biomedical knowledge, small molecule screening strategies and improved chemical methods for synthesis and construction of sophisticated small molecules. Here, we review the mechanisms of post-transcriptional gene regulation, specifically in leukaemia, current small-molecule based efforts to target RNA binding proteins, and future prospects.
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series RNA Biology
spelling doaj-art-37f8ee50b32748519cd1e2eefcd9acb62025-08-20T02:50:03ZengTaylor & Francis GroupRNA Biology1547-62861555-85842024-12-0121127628910.1080/15476286.2024.2303558Small molecule inhibition of RNA binding proteins in haematologic cancerAmit K. Jaiswal0Michelle L. Thaxton1Georgia M. Scherer2Jacob P. Sorrentino3Neil K. Garg4Dinesh S. Rao5Department of Pathology and Laboratory Medicine, University of California, Los Angeles, CA, USADepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA, USADepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA, USADepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA, USADepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA, USADepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA, USAIn recent years, advances in biomedicine have revealed an important role for post-transcriptional mechanisms of gene expression regulation in pathologic conditions. In cancer in general and leukaemia specifically, RNA binding proteins have emerged as important regulator of RNA homoeostasis that are often dysregulated in the disease state. Having established the importance of these pathogenetic mechanisms, there have been a number of efforts to target RNA binding proteins using oligonucleotide-based strategies, as well as with small organic molecules. The field is at an exciting inflection point with the convergence of biomedical knowledge, small molecule screening strategies and improved chemical methods for synthesis and construction of sophisticated small molecules. Here, we review the mechanisms of post-transcriptional gene regulation, specifically in leukaemia, current small-molecule based efforts to target RNA binding proteins, and future prospects.https://www.tandfonline.com/doi/10.1080/15476286.2024.2303558RNA binding proteinspost-transcriptional gene regulationdrug discoveryAcute leukaemiaRNA splicing
spellingShingle Amit K. Jaiswal
Michelle L. Thaxton
Georgia M. Scherer
Jacob P. Sorrentino
Neil K. Garg
Dinesh S. Rao
Small molecule inhibition of RNA binding proteins in haematologic cancer
RNA Biology
RNA binding proteins
post-transcriptional gene regulation
drug discovery
Acute leukaemia
RNA splicing
title Small molecule inhibition of RNA binding proteins in haematologic cancer
title_full Small molecule inhibition of RNA binding proteins in haematologic cancer
title_fullStr Small molecule inhibition of RNA binding proteins in haematologic cancer
title_full_unstemmed Small molecule inhibition of RNA binding proteins in haematologic cancer
title_short Small molecule inhibition of RNA binding proteins in haematologic cancer
title_sort small molecule inhibition of rna binding proteins in haematologic cancer
topic RNA binding proteins
post-transcriptional gene regulation
drug discovery
Acute leukaemia
RNA splicing
url https://www.tandfonline.com/doi/10.1080/15476286.2024.2303558
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