Target intelligence / Profile preview

Small Cajal body-specific RNA 20 (SCARNA20)

Target
SCARNA20
Molecular classification
Small nucleolar RNA (snoRNA), H/ACA box snoRNA, Small Cajal body-specific RNA (scaRNA), Non-coding RNA
01

Overview

Small Cajal body-specific RNA 20 (SCARNA20, also known as ACA66) is a member of the H/ACA box class of small nucleolar RNAs that localizes to Cajal bodies in the nucleus. It functions as a guide RNA, directing the pseudouridylation of uridines within spliceosomal RNAs, primarily guiding modification of residue U28 in U12 minor spliceosomal RNA. SCARNA20 plays a crucial role in the post-transcriptional modification events necessary for proper mRNA splicing and has been shown to be upregulated during cardiomyocyte differentiation, where it promotes proper cardiac gene expression and contractile function. Knockdown of SCARNA20 disrupts these processes, leading to impaired cardiac differentiation. Despite these roles, SCARNA20 is not a protein-coding gene, receptor, or typical pharmacological target, and no drugs directly interact with or modulate its function.

Other names
ACA66scaRNA20
02

Mechanism of action

N/A (no drugs currently target SCARNA20)

03

Biological functions

Guides pseudouridylation (conversion of uridine to pseudouridine) of snRNA, specifically U12 snRNA at residue U28Involved in RNA processing, particularly modification of spliceosomal RNAsPlays a significant role in the regulation of mRNA splicing, especially during cardiomyocyte differentiation and cardiac developmentLocalized to Cajal bodies and nucleolus
04

Disease associations

Cardiac development: Modulation of scaRNA20 impacts cardiac gene expression and cardiomyocyte differentiation; knockdown leads to defective differentiation and altered contractilityAssociated (genomically) with syndromes such as Chromosome 17q23.1–q23.2 deletion syndrome and pontocerebellar hypoplasia, type 7, though direct mechanistic links are not established
05

Safety considerations

Not applicable; as a non-coding RNA, not a therapeutic drug target and no safety data exist
06

Biomarkers

Changes in scaRNA20 expression levels may be used as a biomarker for cardiac differentiation potency or defects in stem cell-derived cardiomyocyte systems

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