Drug intelligence / Profile preview

Sam68 shRNA

Development stage
Preclinical
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Intratumoral, Intraperitoneal, Intravitreal, Inhalation, Ophthalmic, Parenteral
01

Overview

Sam68 shRNA is a research-grade short hairpin RNA (shRNA) construct designed to silence the expression of the Sam68 protein (Src-Associated in Mitosis of 68 kDa), which is encoded by the *KHDRBS1* gene. Sam68 is a multifunctional RNA-binding protein that plays critical roles in RNA processing, alternative splicing, and signal transduction pathways associated with cell cycle regulation and DNA repair. In laboratory settings, Sam68 shRNA is used as a molecular biology tool to investigate the protein's involvement in various pathologies, particularly oncology. Research has demonstrated that knocking down Sam68 in glioblastoma cells impairs DNA double-strand break repair and increases sensitivity to radiation, likely by disrupting the interaction between Sam68 and PARP1. While it is widely used in academic research and available through reagent suppliers like Sigma-Aldrich, it is not currently a commercial therapeutic agent or part of a formal clinical development program.

Other names
KHDRBS1 shRNAKHDRBS-1 shRNAKHDRBS 1 shRNASrc-associated substrate during mitosis of 68 kDa shRNAshSam68shSam-68shSam 68
02

Targets

KHDRBS1

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