Drug intelligence / Profile preview

NTC-AAV

Development stage
Preclinical
Modality
Gene Therapies
Administration
Intrathecal
01

Overview

NTC-AAV (Non-Targeting Control Adeno-Associated Virus) is a research-grade viral vector used as a negative control in gene therapy and CRISPR-based experiments. In the context of Dravet Syndrome research, it is utilized to establish a baseline for the physiological and behavioral effects of AAV-mediated delivery and the expression of CRISPR activation (CRISPRa) components, such as dCas9, without inducing the expression of a specific target gene like *Psd3*. This allows researchers to distinguish the therapeutic effects of a target-specific AAV from the inherent effects of the viral vector and the surgical administration process. It typically contains a non-targeting guide RNA (gRNA) or a non-functional sequence to account for the effects of the viral vector delivery and the presence of the CRISPR machinery without affecting the expression of specific target genes.

Other names
non-target-AAV controlnon-targeting control AAV

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