AAV2-shVMAT2

AAV2-shVMAT2 is a gene therapy experimental agent based on an adeno-associated virus serotype 2 (AAV2) vector encoding a short hairpin RNA (shRNA) designed to silence expression of vesicular monoamine transporter 2 (VMAT2). VMAT2 is a protein responsible for transporting monoamines, such as dopamine, into synaptic vesicles within neurons, facilitating their regulated release. By targeting VMAT2, AAV2-shVMAT2 can modulate dopaminergic signaling, which is of interest in neurological disorder models, particularly in relation to Parkinson’s disease and dopaminergic system function. The modality uses RNA interference to decrease VMAT2 activity, potentially leading to altered dopamine storage and release. This approach aims to manipulate dopamine neurotransmission for research or therapeutic purposes in disorders characterized by dopaminergic dysregulation.[1]

Other names

adeno-associated virus serotype 2 vector encoding short hairpin RNA targeting vesicular monoamine transporter 2