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AAV9-shCOMMD1

Development stage: Unknown
Modality: Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration: Intravenous

Overview

AAV9-shCOMMD1 is a gene therapy candidate based on adeno-associated virus serotype 9 (AAV9) vector technology carrying a short hairpin RNA (shRNA) targeting the COMMD1 gene. The therapy is designed to reduce COMMD1 expression, which may have therapeutic applications in conditions related to cholesterol homeostasis and atherosclerosis based on the known role of COMMD1 in regulating low-density lipoprotein and cholesterol metabolism[6]. AAV9 vectors have demonstrated ability to cross the blood-brain barrier and achieve widespread central and peripheral biodistribution[4].

Targets

COMMD1 (COMM domain-containing protein 1)

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