AAV-Scr-shRNA

AAV-Scr-shRNA is a control gene therapy vector utilized in preclinical research, particularly in studies investigating RNA interference (RNAi) for neurodegenerative conditions like Parkinson's disease. The vector consists of an adeno-associated virus (AAV) capsid containing an expression cassette for a 'scrambled' short-hairpin RNA (shRNA)—a sequence designed to lack homology with any known genomic sequences, ensuring that it does not target or silence any specific gene. In experimental settings, AAV-Scr-shRNA serves as a critical negative control to distinguish the specific therapeutic effects of a target-silencing shRNA from the non-specific cellular responses induced by the viral delivery vehicle or the activation of the RNA-induced silencing complex (RISC). In the context of Parkinson's disease research, it has been used to establish the baseline of disease progression in non-human primate models.

Other names

AAV-scrambled-shRNAscrambled shRNA AAV vector