Adeno-associated viruses (AAV) [1] are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family …

Jun 25, 2025 · We developed a gene therapy, GC301, that delivers a codon-optimized gene encoding human acid α-glucosidase (GAA) and is mediated by recombinant AAV9 (rAAV9 …

Given that AAV9/no MAAP resulted in a significant increase in colocalization with AAV9-CBA-mCherry (Fig. 3C) and the relative GFP mRNA levels (Fig. 4A), we further assessed the …

Apr 29, 2025 · Adeno-associated virus serotype 9 (AAV9) has become a leading vector in gene therapy, providing promising treatments for previously untreatable genetic disorders. Its high …

Adeno-associated virus 9 (AAV9) is a viral vector that can cross the blood-brain barrier and efficiently infect motor neurons, making it a promising candidate for gene therapy applications, …

Jul 26, 2025 · This study explores the potential of adeno-associated virus serotype 9 (AAV9) to deliver therapeutic genes directly into the memory circuit throughout the olfactory bulb (OB), a …

Jan 7, 2026 · Type 1 short QT syndrome (SQT1) is a genetic channelopathy caused by gain-of-function variants in KCNH2, resulting in shortened cardiac repolarization and QT intervals, …

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 …

Oct 30, 2024 · The following experiments identified that the AAV9 MAAP protein exerts a significant influence on in vivo transgene expression. This finding could further explain how …

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 …