Apr 3, 2025 · Jo urn al Pre- pro of 1 Title: AAV Vector Development, Back to the Future 1 Author(s): Lester Suarez-Amaran1,2,4, Liujiang Song1,4, Anna P. Tretiakova1,4, Sheila A Mikhail1, 2 Richard Jude Samulski1,3,* 3 Affiliations & Notes 4 1 M34, Inc. 870 Martin Luther King Jr. Blvd., Chapel Hill, NC 27514-2600, USA. 5 2 Orthopaedic Research Center, C ...

Apr 5, 2025 · We examine the molecular evolution of AAV, detailing advancements in vector engineering, rational design, directed evolution platforms, and computational modeling, which have expanded its therapeutic potential across diverse disease areas.

Apr 3, 2024 · Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different...

Apr 3, 2025 · We examine the molecular evolution of AAV, detailing advancements in vector engineering, rational design, directed evolution platforms, and computational modeling, which have expanded its therapeutic potential across diverse disease areas.

AAV vectors have two main components: the capsid, whose structure varies among AAV serotypes and determines what tissue and cell to target (tropism), and a genome containing a promoter and a transgene. ... and the back of the neck is sanitized. A needle attached to a syringe is manually guided into the cisterna magna, with correct placement ...

May 10, 2023 · Over 15 years after hepatotoxicity was first observed following administration of an adeno-associated virus (AAV) vector during a hemophilia B clinical trial, recent reports of...

Dec 30, 2020 · We find that AAV6 using a CAG promoter to drive transgene expression, delivered into the NP of murine caudal discs at a titer of 10 11 GC/mL, provides excellent transduction efficiency/kinetics and low toxicity in vivo.

Aug 18, 2004 · Adeno-associated virus (AAV) was discovered almost 40 years ago [1–3] and the first AAV vectors were described 20 years ago [4–6], which led to initiation of the first human clinical trial of an AAV vector some 8 years ago, in cystic fibrosis patients [7].

In this regard, recombinant adeno-associated viral vectors (herein referred to as AAV) are powerful tools that can be used both to target and manipulate specific neuronal subtypes (defined based on gene expression, location, and connectivity) and non-neuronal cell types within the nervous system.

Mar 21, 2025 · Adeno-associated virus (AAV) has emerged as a leading platform for gene therapy, enabling the delivery of therapeutic DNA to target cells. However, the potential of AAV to deliver protein payloads has been unexplored. In this study, we engineered a protein carrier AAV (pcAAV) to package and deliver proteins by inserting binding domains on the interior capsid surface. These binding domains ...