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AAV-ARIH2
- Development stage
- Preclinical
- Lead developer
- Icahn School of Medicine
- Modality
- Gene Therapies
- Administration
- Intranasal
01
Overview
AAV-ARIH2 is an experimental adeno-associated virus (AAV)-based gene therapy designed to deliver the host E3 ubiquitin ligase ARIH2 (ariadne RBR E3 ubiquitin ligase 2) to the respiratory tract. Developed as a host-directed therapeutic strategy against Influenza A virus (IAV) infection, AAV-ARIH2 counteracts the viral suppression of ARIH2. During IAV infection, the viral nucleoprotein (NP) selectively suppresses ARIH2 expression, which stabilizes the proviral, pro-apoptotic protein Bcl-2-interacting killer (BIK) and facilitates viral replication. By restoring ARIH2 expression via an AAV6.2 vector, AAV-ARIH2 promotes the K48-linked ubiquitination and proteasomal degradation of BIK, thereby reducing viral load, mitigating lung inflammation, and improving survival.
- Other names
- AAV-mediated ARIH2
02
Targets
E3 ligase (E3 ubiquitin ligases)BIKNP (Taï Forest ebolavirus nucleoprotein)26S proteasome
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