Article ; Online: Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications.
2022 Volume 32, Issue 3, Page(s) 123–138
Abstract: Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic approaches have been developed to correct aberrant alternative splicing events for the treatment of cancers and hereditary diseases, including gene therapy and ... ...
Abstract | Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic approaches have been developed to correct aberrant alternative splicing events for the treatment of cancers and hereditary diseases, including gene therapy and redirecting splicing, using small molecules or splice switching oligonucleotides (SSO). Significant advances in the chemistry and pharmacology of nucleic acid have led to the development of clinically approved SSO drugs for the treatment of spinal muscular dystrophy and Duchenne muscular dystrophy (DMD). In this review, we discuss the mechanisms of SSO action with emphasis on "less common" approaches to modulate alternative splicing, including bipartite and bifunctional SSO, oligonucleotide decoys for splice factors and SSO-mediated mRNA degradation |
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MeSH term(s) | Alternative Splicing/genetics ; Humans ; Muscular Dystrophy, Duchenne/drug therapy ; Muscular Dystrophy, Duchenne/genetics ; Oligonucleotides/genetics ; Oligonucleotides/pharmacology ; Oligonucleotides/therapeutic use ; Oligonucleotides, Antisense/genetics ; Oligonucleotides, Antisense/metabolism ; Oligonucleotides, Antisense/therapeutic use ; RNA Splicing/genetics |
Chemical Substances | Oligonucleotides ; Oligonucleotides, Antisense |
Language | English |
Publishing date | 2022-02-14 |
Publishing country | United States |
Document type | Journal Article ; Review ; Research Support, Non-U.S. Gov't |
ZDB-ID | 2639888-6 |
ISSN | 2159-3345 ; 2159-3337 |
ISSN (online) | 2159-3345 |
ISSN | 2159-3337 |
DOI | 10.1089/nat.2021.0067 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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