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MCOLN1 siRNA
- Development stage
- Preclinical
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Intratumoral
01
Overview
MCOLN1 siRNA is a small interfering RNA designed to silence the expression of the MCOLN1 gene, which encodes the Transient Receptor Potential Mucolipin 1 (TRPML1) protein. TRPML1 is a lysosomal cation channel that plays a critical role in endolysosomal trafficking, autophagy, and cellular homeostasis. In bladder cancer (BLCA), MCOLN1 expression is often inversely correlated with p53 activity and is significantly elevated in tumors harboring TP53 mutations. Research indicates that silencing MCOLN1 using siRNA in p53-deficient bladder cancer cells (such as the T24 line) curtails cell proliferation, induces G0/G1 cell cycle arrest, and reduces the expression of pro-tumorigenic and pro-inflammatory cytokines like IL-6 and TNF-alpha. This suggests that MCOLN1 acts as a permissive factor for cancer cell growth and inflammation in specific genetic backgrounds, making it a potential therapeutic target for p53-mutated malignancies.
- Other names
- TRPML1 siRNATRPML-1 siRNATRPML 1 siRNAMucolipin-1 siRNAMucolipin1 siRNAMucolipin 1 siRNAsiMCOLN1siMCOLN-1siMCOLN 1
02
Targets
MCOLN1 (Transient receptor potential cation channel mucolipin subfamily member 1)
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