GEN spoke with experts in this field for their input as to how they are working to overcome issues to optimize AAV gene delivery. For example, to address targeting challenges, companies are ...

and other AAV serotypes to identify novel capsids optimized for AAV delivery for specific therapeutic applications. Voyager’s next-generation capsids are designed to deliver innovative AAV-based ...

3 Therefore, targeted gene therapies need a tailored vector to enhance the specificity and transgene delivery efficiency to target organs, tissues or cell types. Existing AAV transgene delivery ...

Safe and effective gene therapy strategies depend on robust and reliable adeno-associated virus (AAV) mediated therapeutic gene delivery. However, scaling up AAV manufacturing to obtain sufficient ...

“This exploratory research represents our forward-thinking approach within our diabetes program to determine if our novel diabetes gene therapy could be delivered using a non-viral delivery system, ...

(6) Researchers analyze NGS data to study patterns of viral DNA expression, which may identify AAV that have a trophism for certain tissues, and may thus serve as better gene delivery vehicles when ...

While AAV delivery is a well understood delivery mechanism, there are many benefits to a non-viral delivery system, including the potential for re-dosing patients to optimize treatment.

In a recent review published in Signal Transduction and Targeted Therapy, researchers presented recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

To permanently access this Technical Note for free, fill out the short form below. Adeno-associated virus (AAV) is a prominent method for delivering genes in vivo. Therapeutic delivery to target cells ...

"As a novel approach to treat chronic hypereosinophilia, we hypothesized that AAV-mediated delivery of an anti-human eosinophil antibody would provide one-time therapy that would mediate ...

AI-assisted AAV capsid engineering has transformative potential in terms of accelerating and optimizing the development of gene therapies.