AAV-AFG3L2

AAV-AFG3L2 is an experimental gene therapy construct designed to deliver and overexpress the AFG3L2 (AFG3-like protein 2) gene using an adeno-associated virus (AAV) vector. AFG3L2 encodes a mitochondrial m-AAA protease that is essential for mitochondrial protein quality control and the assembly of the oxidative phosphorylation system. In preclinical models of Crohn's disease and experimental colitis, AAV-AFG3L2-mediated overexpression has been shown to protect intestinal epithelial cells from ferroptosis and oxidative stress. The therapeutic mechanism involves the activation of the PPARA (Peroxisome proliferator-activated receptor alpha) signaling pathway, which subsequently upregulates the transcription of GPX4 (Glutathione peroxidase 4), a key antioxidant enzyme that prevents lipid peroxidation and ferroptotic cell death. By restoring mitochondrial ultrastructure and enhancing antioxidant capacity, AAV-AFG3L2 aims to maintain intestinal mucosal integrity and reduce inflammation.

Other names

AFG3L2 gene therapyAFG-3L2 gene therapyAFG 3L2 gene therapyAAV-mediated AFG3L2 overexpression