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CD4 aptamer-siRNA
- Development stage
- Preclinical
- Lead developer
- Boston Children's Hospital
- Modality
- RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, 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, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intraperitoneal, Intravenous, Topical, Vaginal
01
Overview
CD4 aptamer-siRNA chimeras (CD4-AsiCs) are a class of targeted RNA therapeutics designed to deliver small interfering RNAs (siRNAs) specifically to CD4-expressing cells, such as helper T cells and macrophages. This platform utilizes a CD4-binding RNA or DNA aptamer covalently or non-covalently linked to a therapeutic siRNA payload. Upon binding to the CD4 receptor, the chimera is internalized via receptor-mediated endocytosis, and the siRNA is processed by the endogenous Dicer enzyme to silence target genes. CD4-AsiCs have been investigated for various therapeutic applications, including the prevention of HIV transmission (by targeting viral genes like gag and vif, or host co-receptors like CCR5) and cancer immunotherapy (by targeting PITPNM3 to block regulatory T cell recruitment in breast cancer).
- Other names
- CD4 aptamer-siRNA chimeraCD-4 aptamer-siRNA chimeraCD 4 aptamer-siRNA chimeraCD4 aptamer-siRNA chimerasCD-4 aptamer-siRNA chimerasCD 4 aptamer-siRNA chimerasCD4-AsiCCD-4-AsiCCD 4-AsiCCD4-AsiCsCD-4-AsiCsCD 4-AsiCs
02
Targets
Human immunodeficiency virus type 1 gag messenger RNARORγtHuman immunodeficiency virus type 1 virion infectivity factor messenger RNA (HIV-1 vif mRNA)CD4 (T lymphocyte surface antigen)
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