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GNAS stimRNA
- Development stage
- Preclinical
- Lead developer
- Thomas Jefferson University
- 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
01
Overview
GNAS stimRNA is a platelet-selective, naked short inhibitory modified RNA (stimRNA) designed to suppress the expression of Gnas (encoding the Gs alpha subunit) [PB3012]. Developed by researchers at Thomas Jefferson University, it targets the prostacyclin (PGI2) pathway in platelets [PB3012]. By knocking down Gnas, the stimRNA counteracts the inhibitory effects of prostacyclin on platelet activation, thereby enhancing platelet aggregation and improving hemostasis [PB3012]. It is being investigated for the control of acquired bleeding, particularly bleeding complications associated with antiplatelet therapies such as ticagrelor [PB3012].
- Other names
- Gnas stimRNAnaked GNAS stimRNA
02
Targets
AC (Adenylyl cyclase family)AGO2 (Argonaute RISC catalytic component 2)GNAS
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