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AAV2-CAG-interferon beta
- Development stage
- Preclinical
- Modality
- Gene Therapies
- Administration
- Ophthalmic
01
Overview
AAV2-CAG-interferon beta is an adeno-associated virus (AAV) gene therapy construct utilizing the serotype 2 (AAV2) capsid and the strong, constitutive CAG promoter to drive the expression of interferon beta (IFN-β). In the context of ophthalmic research, particularly for age-related macular degeneration (AMD), this vector is used to model the effects of chronic IFN-β overexpression in the retina. Studies have demonstrated that while the STING/IFN-β pathway may provide initial cytoprotection, sustained AAV2-mediated overexpression of IFN-β leads to metabolic and phototransduction dysregulation, ultimately promoting photoreceptor apoptosis and choroidal disruption. This construct serves as a critical tool for investigating the transition of the STING pathway from a protective immune surveillance mechanism to a pathogenic driver of retinal degeneration.
- Other names
- AAV2-CAG-IFN-βAAV2-mediated IFN-β
02
Targets
IFNAR (Immune system modulation via type I interferon receptor)
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