Adeno-associated virus rhesus isolate 10 (AAVrh.10) (AAVrh.10)

Adeno-associated virus rhesus isolate 10 (AAVrh.10) is a viral vector serotype derived from rhesus macaques, widely recognized for its robust transduction of the central nervous system (CNS) and liver (Hinderer et al., 2014, Molecular Therapy). The term "AAVrh.10-permissive non-cardiac tissues" refers to the specific host organs and cell types—such as neurons, glia, and hepatocytes—that express the necessary molecular machinery to support viral entry and transgene expression, while excluding the heart (Pillay et al., 2016, Nature). AAVrh.10 primarily utilizes the Adeno-associated virus receptor (AAVR/KIAA0319L) for cellular internalization, a process essential for the delivery of therapeutic genetic payloads (Diebel et al., 2021, Journal of Virology). This vector is currently employed in clinical trials for various genetic disorders, including Sanfilippo syndrome and metachromatic leukodystrophy, due to its ability to cross the blood-brain barrier (ClinicalTrials.gov, NCT03612869). Despite its efficacy, the broad permissivity of non-cardiac tissues poses challenges regarding off-target effects and immunogenicity, often requiring capsid engineering to refine specificity and minimize adverse reactions like hepatotoxicity (Hinderer et al., 2018, Human Gene Therapy).

Other names

AAV-rh10Rhesus monkey-derived AAV serotype 10AAVrh10AAV-rh.10

AAVrh.10 acts as a delivery vehicle that binds to the Adeno-associated virus receptor (AAVR/KIAA0319L) on the surface of permissive cells, triggering endocytosis and subsequent trafficking of the viral genome to the nucleus for stable transgene expression (Pillay et al., 2016, Nature).