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 …

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.

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.

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 – …

AAV9 is an efficient natural adeno-associated virus (AAV) serotype for transgene expression in neurons. Surprisingly, the AAV9 serotype produced wide-scale neuronal transduction in the CNS from a peripheral, systemic injection in neonatal subjects.

Mar 18, 2025 · Explore the key properties of the AAV9 capsid that influence its distribution, stability, and transport for effective brain-wide gene delivery. Adeno-associated virus serotype 9 (AAV9) is a powerful gene therapy tool, known for its ability to cross the blood-brain barrier and distribute widely in the central nervous system.

Isolated originally by James Wilson and colleagues, AAV9 is one of a very large family of AAV clades containing more than 100 new serotypes that remain poorly characterized. 7,8 Of these, AAV8 and AAV9 stand out; vectors carrying capsids from these serotypes transduce rodent muscle, liver, and lung about 100-fold more efficiently than AAV2.

There are numerous channels that actively transport important molecules from the blood into the brain that might be candidate AAV9 receptors. Several candidates are depicted, but this is not a complete list of possible transporters.