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AAV2-CAG-Null

Development stage
Unknown
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration
Ophthalmic
01

Overview

AAV2-CAG-Null is a recombinant adeno-associated virus (AAV) vector of serotype 2 that utilizes the strong, constitutive CAG promoter but lacks a functional transgene (a "null" or "empty" vector). In preclinical research, particularly in ophthalmology and gene therapy studies, it serves as a critical negative control to distinguish the biological effects of the viral vector capsid, the promoter activity, and the administration procedure from the specific effects of a therapeutic gene. The CAG promoter is a synthetic construct comprising the cytomegalovirus (CMV) early enhancer, the chicken beta-actin promoter, and the rabbit beta-globin splice acceptor, ensuring high levels of expression in various mammalian tissues. In the context of retinal research, such as studies on age-related macular degeneration (AMD), this vector is typically administered via intravitreal injection to provide a baseline for comparison against vectors expressing therapeutic proteins like IFN-β.

Other names
AAV2-CAG-EmptyAAV-2-CAG-EmptyAAV 2-CAG-EmptyAAV2-CAG-ControlAAV-2-CAG-ControlAAV 2-CAG-Control
02

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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