Small nucleolar RNA SNORA70 (SNORA70)

Small nucleolar RNA SNORA70 is a non-coding RNA of the H/ACA-box snoRNA subfamily, which serves as a guide for posttranscriptional pseudouridylation of ribosomal RNA and other target RNAs in the nucleolus. SNORA70 is crucial for proper cell differentiation in muscle progenitor cells and is notably downregulated in myotonic dystrophy type 1, contributing to the impaired differentiation phenotype by regulating modification of the CCND3 mRNA. The snoRNA’s action is largely posttranscriptional, relying on host gene splicing for its maturation, and altered SNORA70 levels may represent a biomarker or investigational target for diseases involving splicing defects. While snoRNAs are not classical drug targets, gene and RNA-based therapies are being studied to modulate their activity for disease correction.

Other names

SNORA70LOC124900261H/ACA box snoRNA SNORA70

Antisense oligonucleotides (experimental): In cell models, antisense oligonucleotides (ASOs) directed against SNORA70 binding sites on target RNAs can block its function, preventing pseudouridylation and destabilizing target transcripts (e.g., CCND3 mRNA). No small molecules, antibodies, or other conventional drug modalities are described.