Therapeutic modulation of RNA splicing in malignant and non-malignant disease Review
| Authors: | El Marabti, E.; Abdel-Wahab, O. |
| Review Title: | Therapeutic modulation of RNA splicing in malignant and non-malignant disease |
| Abstract: | RNA splicing is the enzymatic process by which non-protein coding sequences are removed from RNA to produce mature protein-coding mRNA. Splicing is thereby a major mediator of proteome diversity as well as a dynamic regulator of gene expression. Genetic alterations disrupting splicing of individual genes or altering the function of splicing factors contribute to a wide range of human genetic diseases as well as cancer. These observations have resulted in the development of therapies based on oligonucleotides that bind to RNA sequences and modulate splicing for therapeutic benefit. In parallel, small molecules that bind to splicing factors to alter their function or modify RNA processing of individual transcripts are being pursued for monogenic disorders as well as for cancer. © 2021 The Author(s) |
| Keywords: | rna; myelodysplastic syndromes; splicing; spinal muscular atrophy; antisense oligonucleotides; muscular dystrophy |
| Journal Title: | Trends in Molecular Medicine |
| Volume: | 27 |
| Issue: | 7 |
| ISSN: | 1471-4914 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2021-07-01 |
| Start Page: | 643 |
| End Page: | 659 |
| Language: | English |
| DOI: | 10.1016/j.molmed.2021.04.005 |
| PUBMED: | 33994320 |
| PROVIDER: | scopus |
| PMCID: | PMC8238868 |
| DOI/URL: | |
| Notes: | Review -- Export Date: 2 August 2021 -- Source: Scopus |
