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tRNA splicing endonuclease subunit 15 (TSEN15) mRNA 3' untranslated region (UTR) (TSEN15 mRNA 3' UTR)
- Target
- TSEN15 mRNA 3' UTR
- Molecular classification
- RNA, Untranslated region
01
Overview
The tRNA splicing endonuclease subunit 15 (TSEN15) mRNA 3' untranslated region (UTR) is a critical regulatory domain of the transcript encoding the TSEN15 protein, a subunit of the heterotetrameric tRNA splicing endonuclease complex (UniProt Q8WW01). This complex is essential for the removal of introns from precursor tRNAs, a fundamental step in protein synthesis (PubMed: 25224756). The 3' UTR contains cis-acting elements that regulate mRNA stability and translation, making it a strategic target for therapeutic modulation. Mutations in TSEN15 are the primary cause of Pontocerebellar Hypoplasia Type 2F (PCH2F), a severe neurodevelopmental disorder characterized by cerebellar and pontine atrophy (OMIM: 615759). Therapeutic strategies targeting this region, such as antisense oligonucleotides (ASOs), aim to increase the expression of functional TSEN15 protein by blocking inhibitory regulatory elements or microRNA binding sites. This approach is designed to restore tRNA processing capacity and potentially mitigate the neurodegenerative progression seen in affected individuals.
- Other names
- C1orf19 mRNA 3' UTRSEN15 mRNA 3' UTRtRNA-intron endonuclease subunit SEN15 mRNA 3' UTRtRNA splicing endonuclease subunit 15 mRNA 3' untranslated region
02
Mechanism of action
Modulation of mRNA stability or translation efficiency via steric hindrance of regulatory elements
03
Biological functions
RNA stability regulationTranslation regulationtRNA processing
04
Disease associations
Pontocerebellar hypoplasia type 2FNeurodevelopmental disorder
05
Safety considerations
Off-target RNA bindingInflammatory response to oligonucleotidesOverexpression toxicity
06
Interacting drugs
Antisense oligonucleotides (experimental)
07
Biomarkers
TSEN15 protein levelstRNA splicing efficiencyCerebellar volume via MRI
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