Drug intelligence / Profile preview

Smarcc1 shRNA

Development stage
Preclinical
Lead developer
University of Freiburg
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

Smarcc1 shRNA is a preclinical research asset and potential therapeutic modality designed to target and silence the **SMARCC1** (SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily C member 1) gene. SMARCC1 is a core subunit of the **SWI/SNF (BAF) chromatin remodeling complex**, which is essential for regulating gene expression. Research presented at ASH 2024 identified SMARCC1 as a critical molecular dependency in **T-cell acute lymphoblastic leukemia (T-ALL)**, particularly in cases driven by oncogenic **NOTCH1** signaling. The shRNA functions via the RNA interference (RNAi) pathway to degrade SMARCC1 mRNA, thereby reducing protein levels and disrupting the SMARCC1-NOTCH1 interaction. In experimental models, Smarcc1 knockdown has been shown to induce apoptosis, inhibit leukemia cell proliferation, and significantly decrease leukemic infiltration in vivo, suggesting that SMARCC1 is a viable epigenetic target for the treatment of NOTCH1-dependent malignancies.

Other names
SMARCC1 shRNASMARCC-1 shRNASMARCC 1 shRNASmarcc1-targeting shRNASmarcc-1-targeting shRNASmarcc 1-targeting shRNA
02

Targets

SMARCC1 (SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily C member 1)

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