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AAV-shPank3
- Development stage
- Preclinical
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-shPank3 is an experimental gene therapy vector that utilizes an adeno-associated virus (AAV) to deliver short hairpin RNA (shRNA) targeting Pantothenate Kinase 3 (PANK3). PANK3 is a key enzyme in the Coenzyme A biosynthesis pathway and has been identified as a critical component of the c-Myc-PANK3-EMT axis, which regulates the structural integrity and function of the intestinal barrier. In preclinical models of ulcerative colitis, AAV-shPank3-mediated knockdown of PANK3 has been shown to inhibit epithelial-mesenchymal transition (EMT) and promote the repair of the intestinal epithelium. This approach represents a potential therapeutic strategy for restoring barrier function and treating the underlying pathology of ulcerative colitis by modulating the c-Myc-PANK3-EMT axis.
- Other names
- AAV-shRNA-PANK3AAV-shRNA-PANK-3AAV-shRNA-PANK 3AAV-shPANK3AAV-shPANK-3AAV-shPANK 3
02
Targets
PANK3 (Pantothenate kinase 3)
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