Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae.

Adeno-associated virus (AAV) is a versatile viral vector technology that can be engineered for very specific functionality in gene therapy applications. To date, AAV has been shown to be safe and effective in preclinical and clinical settings.

Feb 10, 2025 · Explore the advancements in AAV9 gene therapy, focusing on its composition, cellular mechanisms, and production techniques. Adeno-associated virus serotype 9 (AAV9) has become a leading vector in gene therapy, providing promising treatments for previously untreatable genetic disorders.

Aug 21, 2019 · Here, we evaluated the safety of cerebellomedullary (CM) cistern injection of adeno-associated viral vector serotype 9 encoding human ASM (AAV9-hASM) in nonhuman primates (NHP). We also evaluated its therapeutic benefit …

May 17, 2024 · We generated a new galactose binding-deficient AAV9 peptide display library and selected two new AAV9 engineered capsids with enhanced targeting in mouse and marmoset brains after intravenous delivery.

Oct 30, 2024 · Recombinant adeno-associated viruses (AAVs) are used extensively in clinical gene therapy for treating a range of tissues and pathologies in humans. In particular, AAV9 occupies a prominent position in central nervous system (CNS) gene therapy given its central role in ongoing clinical trials and an …

May 16, 2011 · Vectors based on adeno-associated virus (AAV) serotype 9 are candidates for in vivo gene delivery to many organs, but the receptor (s) mediating these tropisms have yet to be defined. We evaluated AAV9 uptake by glycans with terminal sialic acids (SAs), a common mode of cellular entry for viruses.

In the present study, we evaluated the ability of AAV9 to transduce primary sensory neurons as well as its distribution to brain regions due to rostral flow of CSF when delivered intrathecally by direct lumbar puncture.

AAV9 is one of the most promising serotypes for gene therapeutic applications because of the wild-type (WT) and recombinant construct capsid characteristics (16 – 19). AAV9 transduces a wide range of tissue types, including cardiac and skeletal muscle, liver, pancreas, and eye (18, 20 – …

Dec 12, 2024 · Gene therapies such as onasemnogene abeparvovec for spinal muscular atrophy (SMA) utilize adeno-associated virus 9 (AAV9) for targeted gene delivery, which requires an AAV9 antibody (AAV9-Ab) immunoglobulin G (IgG) ≤1:50 titer threshold.