AAV8-microRNA-LRP1

AAV8-microRNA-LRP1 is an investigational gene therapy drug composed of a recombinant adeno-associated virus serotype 8 (AAV8) vector engineered to deliver a synthetic microRNA sequence designed to target and silence the expression of the low-density lipoprotein receptor-related protein 1 (LRP1) gene. The mechanism relies on the robust hepatic tropism of the AAV8 vector, facilitating highly efficient liver transduction following systemic administration. Once delivered, the synthetic microRNA guides the RNA-induced silencing complex (RISC) to degrade LRP1 mRNA or suppress its translation, resulting in reduced LRP1 protein levels in target tissues. This approach represents a modality within RNA therapy and gene therapy, primarily studied in preclinical research for diseases where LRP1 modulation is therapeutically relevant, such as neurodegenerative disorders or metabolic liver diseases. Currently, there is limited public reporting of clinical-stage development for this exact construct.