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AAV2-si-IRF-1
- Development stage
- Preclinical
- Lead developer
- Guizhou Provincial People's Hospital
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
AAV2-si-IRF-1 is an adeno-associated virus serotype 2 (AAV2) gene therapy vector designed to deliver a small interfering RNA (siRNA) targeting interferon regulatory factor 1 (IRF-1) [A0915]. In preclinical models of hyperoxaluria and calcium oxalate (CaOx) nephrocalcinosis, AAV2-si-IRF-1 is administered via renal subcapsular injection to downregulate IRF-1 expression in the kidney [A0915]. This downregulation of IRF-1 modulates the IRF-1/miR-301/CSF-1 signaling pathway, promoting the polarization of macrophages from the proinflammatory M1 phenotype to the anti-inflammatory M2 phenotype [A0915]. Consequently, treatment with AAV2-si-IRF-1 reduces intrarenal CaOx crystal deposition and attenuates renal injury, suggesting its potential utility as a therapeutic strategy or research tool for nephrolithiasis and hyperoxaluria-induced kidney damage [A0915].
- Other names
- AAV2-si-IRF1AAV-2-si-IRF1AAV 2-si-IRF1AAV2-si-Irf-1AAV-2-si-Irf-1AAV 2-si-Irf-1
02
Targets
IRF1 (Interferon regulatory factor 1)
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