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AAV9-shENG

Development stage: Preclinical
Modality: Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

AAV9-shENG is an experimental gene therapy construct designed to silence the expression of endoglin (ENG) through RNA interference. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a short hairpin RNA (shRNA) sequence that targets endoglin mRNA for degradation. Endoglin is a type I transmembrane protein and a co-receptor for the transforming growth factor-beta (TGF-β) signaling pathway, which is highly expressed in vascular endothelial cells. In the context of Alzheimer's disease research, AAV9-shENG is used to investigate the pathological role of endothelial endoglin in promoting astrocyte reactivity and neuroinflammation. By knocking down endoglin in brain microvascular endothelial cells, researchers aim to mitigate cerebrovascular dysfunction and its downstream neurodegenerative effects.

Other names: AAV9-shRNA-EndoglinAAV-9-shRNA-EndoglinAAV 9-shRNA-EndoglinAAV9-shRNA-ENGAAV-9-shRNA-ENGAAV 9-shRNA-ENG

Targets

ENG (Integrin Beta 6)

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