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ADNP siRNA
- Development stage
- Preclinical
- Lead developer
- University of Virginia
- 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
- Intrathecal, Intratumoral
01
Overview
ADNP siRNA is an experimental RNA interference-based approach designed to deplete the Activity-Dependent Neuroprotective Protein (ADNP). ADNP is a critical component of the ChAHP complex, which also includes CBX3 and CHD4, and is involved in chromatin remodeling, RNA splicing, and transcriptional regulation. In the context of acute myeloid leukemia (AML), ADNP acts as a scaffold for the recruitment of the ChAHP complex to Lysine Specific Demethylase 1 (LSD1). Research indicates that the depletion of ADNP in erythroleukemia models, such as K562 cells, induces spontaneous erythroid differentiation, inhibits cell proliferation, and promotes apoptosis. This suggests that the LSD1/ADNP axis is a potential therapeutic target for pro-differentiation therapies in AML.
- Other names
- ADNP depletionADNP siRNA/depletion
02
Targets
Activity-dependent neuroprotective protein mRNA (ADNP mRNA)
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