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AAV8MT-miR-199a

Development stage: Preclinical
Modality: Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics

Overview

AAV8MT-miR-199a is a gene therapy utilizing an Adeno-associated virus (AAV) vector, specifically a modified AAV8 serotype (AAV8MT), to deliver microRNA-199a (miR-199a). It is being investigated for its therapeutic potential in hepatocellular carcinoma (HCC). The active component, miR-199a-5p, is known to repress the expression of hypoxia-inducible factor 1α (HIF-1α), a key molecular mediator that contributes to increased resistance to therapy and tumor relapse in HCC. Studies have shown that overexpression of miR-199a-5p alone can significantly decrease HIF-1α mRNA levels and enhance cancer cell cytotoxicity in both in vitro and in vivo xenograft models.

Targets

HIF1A (Hypoxia-inducible factor 1-alpha)

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