Drug intelligence / Profile preview

siCPT1A

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
Experimental Only (typically In Vitro Or Via Local/systemic Injection In Animal Models)
01

Overview

siCPT1A is a **small interfering RNA (siRNA)** designed to specifically silence the expression of the *carnitine palmitoyltransferase 1A (CPT1A)* gene. CPT1A is an enzyme critical for mitochondrial fatty acid oxidation, highly expressed in metabolically active tissues like liver and in certain cancer cell contexts. siCPT1A functions by promoting the degradation of CPT1A mRNA, thus reducing protein production. Research using siCPT1A has shown that **inhibiting CPT1A sensitizes cancer cells to chemotherapy, suppresses cancer stem cell stemness, and reduces the activation of hepatic stellate cells implicated in fibrosis**. These findings suggest that siCPT1A, as a molecular research tool or experimental therapeutic, has potential utility in diseases involving CPT1A overactivity—particularly in oncology (ovarian cancer, liver cancer) and fibrotic disorders[1][2].

Other names
small interfering RNA targeting CPT1A
02

Targets

CPT1A (Carnitine palmitoyltransferase 1A)

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