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High-throughput Screening for Chemical Modulators of Post-transcriptionally Regulated Genes
Published on: March 3, 2015
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Pseudouridylation-mediated gene expression modulation
Jonathan L Chen1, W-Matthias Leeder2, Pedro Morais3
1Department of Biochemistry and Biophysics, Center for RNA Biology, University of Rochester Medical Center, Rochester, NY, U.S.A.
The Biochemical Journal
|January 4, 2024
Summary
RNA-guided pseudouridylation modifies RNA for splicing and premature termination codon readthrough. This review explores its mechanisms, functions, and therapeutic potential in genetic diseases and cancer.
Area of Science:
- Molecular Biology
- RNA Biology
- Biochemistry
Background:
- RNA-guided pseudouridylation is a key post-transcriptional modification.
- It plays crucial roles in pre-mRNA splicing and premature termination codon (PTC) readthrough.
- Understanding its mechanisms is vital for exploring therapeutic applications.
Approach:
- This review examines the molecular mechanisms of RNA-guided pseudouridylation.
- It highlights the roles of guide RNAs and pseudouridine synthases in uridine-to-pseudouridine conversion.
- The impact on U2 small nuclear RNA, pre-mRNA splicing, and PTC readthrough is analyzed.
Key Points:
- RNA-guided pseudouridylation of U2 small nuclear RNA influences spliceosome assembly and branch site recognition.
- This modification regulates PTC readthrough, affecting translation termination and genetic disorders.
- Pseudouridine modifications show promise for treating genetic diseases, cancer, and in mRNA vaccine development.
Conclusions:
- RNA-guided pseudouridylation is a significant regulatory mechanism in RNA processing.
- Its diverse functions offer potential for novel therapeutic strategies.
- Further research can unlock the full potential of pseudouridine modifications in medicine.
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