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AAV9-shIFIT3
- Development stage
- Preclinical
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Rectal
01
Overview
AAV9-shIFIT3 is an experimental gene therapy agent designed to treat ulcerative colitis by silencing the expression of the IFIT3 gene. It consists of an adeno-associated virus serotype 9 (AAV9) vector that delivers a short hairpin RNA (shRNA) expression cassette specifically targeting IFIT3 mRNA. IFIT3 (Interferon-induced protein with tetratricopeptide repeat 3) is a protein involved in antiviral and inflammatory signaling; in the context of ulcerative colitis, its upregulation promotes the polarization of macrophages toward the pro-inflammatory M1 phenotype and activates the STAT1/2 signaling pathway. By knocking down IFIT3, AAV9-shIFIT3 aims to suppress colonic inflammation and restore immune homeostasis. This agent is currently in the preclinical research stage.
- Other names
- AAV9-shRNA-IFIT3AAV-9-shRNA-IFIT3AAV 9-shRNA-IFIT3AAV9-sh-IFIT3AAV-9-sh-IFIT3AAV 9-sh-IFIT3
02
Targets
IFIT3 (Interferon-induced protein with tetratricopeptide repeat 3)
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