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AAV-CRISPR Cx36 knockdown

Development stage: Preclinical
Lead developer: VA Boston Healthcare System
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Parenteral

Overview

AAV-CRISPR Cx36 knockdown is an experimental gene therapy approach designed to selectively reduce the expression of the neuronal gap junction protein connexin36 (Cx36) in specific brain regions, such as the thalamic reticular nucleus (TRN). Utilizing an adeno-associated virus (AAV) vector to deliver CRISPR-Cas components, this tool enables localized gene silencing to study the role of electrical synapses in thalamocortical network oscillations and state regulation. Research indicates that Cx36 knockdown in the TRN impairs task-evoked cortical gamma activity, a biomarker associated with neuropsychiatric disorders like schizophrenia, suggesting Cx36 as a potential therapeutic target for cognitive and oscillatory deficits.

Other names: TRN-Cx36 KDTRN-Cx-36 KDTRN-Cx 36 KDCx36 gene knockdownCx-36 gene knockdownCx 36 gene knockdownAAV-mediated CRISPR Cx36 knockdown

Targets

GJD2 (Gap junction delta-2 protein)

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