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AAV2-shControl

Development stage
Preclinical
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies
Administration
Intracerebral, Intravitreal, Intravenous
01

Overview

AAV2-shControl is an experimental gene therapy reagent utilizing an adeno-associated virus serotype 2 (AAV2) vector to deliver a control short hairpin RNA (shRNA) sequence. The term "shControl" indicates that the construct does not target a specific gene, and is used as a negative control in studies evaluating RNA interference-based gene therapy. The AAV2 vector is widely used in gene therapy due to its neuronal tropism and low immunogenicity. AAV2-shControl serves as a control arm in experimental settings for comparison with therapeutic AAV2-shRNA constructs targeting specific genes. Its primary application is in preclinical and early-stage research to validate safety, specificity, and non-target effects of AAV2-mediated RNA interference methodologies.

Other names
AAV2-shControlAAV-2-shControlAAV 2-shControl
02

Targets

αVβ5 (Integrin αVβ5)ITGA5:ITGB1 (Integrin α5β1)FGFR1 (Fibroblast growth factor receptor 1)HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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