Unspecified AAV-PR host cell-surface receptors on cerebral pericytes and vascular smooth muscle cells

AAV-PR is an engineered adeno-associated virus (AAV) capsid variant derived from AAV9, characterized by the insertion of a specific 7-mer peptide (PRPPSTH) into the VP1 protein at position 588. It was developed to overcome the limitations of natural AAV serotypes in targeting the brain's vascular components, demonstrating a unique and potent tropism for cerebral pericytes and vascular smooth muscle cells (VSMCs) following systemic administration. The 'unspecified AAV-PR host cell-surface receptors' represent the currently uncharacterized molecular targets on these mural cells that facilitate the vector's high-efficiency binding and internalization. While the parent AAV9 capsid primarily targets neurons and astrocytes, the modifications in AAV-PR redirect its entry toward the vasculature, making it a critical tool for treating neurodegenerative and cerebrovascular diseases where vascular dysfunction is a primary driver. Potential therapeutic applications include gene delivery for Alzheimer's disease, Moyamoya disease, and Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS). Research is ongoing to identify the specific receptor(s) involved, which is essential for refining the vector's specificity and ensuring safety in clinical applications.

Other names

AAV-PR receptorsCerebral pericyte AAV receptorsVascular smooth muscle cell AAV receptorsPRPPSTH-binding receptors

AAV-PR binds to these unspecified cell-surface receptors on pericytes and vascular smooth muscle cells to facilitate viral entry and transgene delivery.