AAV9-miRNA

AAV9-miRNA refers to a class of **gene therapy constructs** in which a microRNA (miRNA) is delivered to target tissues using an engineered adeno-associated virus serotype 9 (AAV9) vector. The approach exploits the efficient in vivo gene delivery capability and tissue tropism of AAV9, most notably for brain, heart, skeletal muscle, and other tissues. These constructs can be designed to deliver either miRNA mimics (for overexpression) or miRNA inhibitors (for knockdown). A specific example, scAAV9-miR-23a, has been developed and tested in preclinical models of spinal muscular atrophy (SMA), where it reduced disease severity by increasing motor neuron size, decreasing neuromuscular junction pathology, and inhibiting atrophy-related genes (such as Atrogin1/MuRF1)[2]. The broad mechanism is **post-transcriptional regulation** of gene networks through miRNA activity, which may include the neuroprotection, modulation of apoptosis, and inhibition of muscle atrophy pathways. While the general concept and technology are established in preclinical research, specific AAV9-miRNA constructs are not approved pharmaceuticals and represent an investigational gene/RNA therapy strategy[1][2].

Other names

scAAV9-miRNAscAAV-9-miRNAscAAV 9-miRNArAAV9-miRNArAAV-9-miRNArAAV 9-miRNAAAV9-mediated miRNA deliveryAAV-9-mediated miRNA deliveryAAV 9-mediated miRNA deliveryAAV9-miR-23aAAV-9-miR-23aAAV 9-miR-23a