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 ...
However, system upgrades to the leading gene delivery vehicle offer hope that effective therapies will soon be within reach. There is a growing list of approved AAV-based gene therapies and hundreds ...
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 ...
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 ...
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Gene therapy could treat chronic hypereosinophilia"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 ...
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 ...
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.
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Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdlesIn a recent review published in Signal Transduction and Targeted Therapy, researchers presented recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.
The axolotl (Ambystoma mexicanum) has extraordinary regenerative abilities—the salamander can regrow lost limbs and repair ...
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