Drug intelligence / Profile preview

shRNA-SON

Development stage
Preclinical
Lead developer
Tokyo Women's Medical University
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

shRNA-SON is a short hairpin RNA (shRNA) vector designed to target and knockdown the expression of the SON DNA-binding protein. SON is a large protein involved in various cellular processes, including RNA splicing and cell cycle regulation. In the context of pancreatic cancer, SON has been identified as a novel therapeutic target. Knockdown of SON using shRNA-SON has been shown to induce G2/M cell cycle arrest and apoptosis in pancreatic cancer cells, while having minimal effect on normal pancreatic duct epithelial cells. Preclinical studies in xenograft models demonstrated that stable expression of shRNA-SON significantly retards tumor formation.

02

Targets

SON (Transcription factor SON)

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