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Adeno-associated virus as a delivery vector for gene therapy of human diseases

739
Citations
April 3, 2024
Published Date

Research Abstract & Technology Focus

AbstractAdeno-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 tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods. Seven rAAV-based gene therapy products have received regulatory approval, but there continue to be concerns about safely using high-dose viral therapies in humans, including immune responses and adverse effects such as genotoxicity, hepatotoxicity, thrombotic microangiopathy, and neurotoxicity. In this review, we explore AAV biology with an emphasis on current vector engineering strategies and manufacturing technologies. We discuss how rAAVs are being employed in ongoing clinical trials for ocular, neurological, metabolic, hematological, neuromuscular, and cardiovascular diseases as well as cancers. We outline immune responses triggered by rAAV, address associated side effects, and discuss strategies to mitigate these reactions. We hope that discussing recent advancements and current challenges in the field will be a helpful guide for researchers and clinicians navigating the ever-evolving landscape of rAAV-based gene therapy.
adeno-associated virus delivery vector therapy human
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What is the core focus of the research titled 'Adeno-associated virus as a delivery vector for gene therapy of human diseases'?

This literature focuses on: AbstractAdeno-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 tissues. Recombinant AAV (rAAV) has been engine...

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What other academic literature is closely related to 'Adeno-associated virus as a delivery vector for gene therapy of human diseases'?

Yes, highly correlated activity was mapped. An entry titled 'Adeno-associated virus as a delivery vector for gene therapy of human diseases' discusses this: AbstractAdeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability...

Are there commercial applications of 'Adeno-associated virus as a delivery vector for gene therapy of human diseases' in market news publications?

Yes, highly correlated activity was mapped. An entry titled 'Modulating nuclear stiffness and envelope barrier facilitates AAV nuclear entry and reduces immunogenicity' discusses this: Low efficiency and immunotoxicity limit rAAVs clinical use. Here, the authors show that encapsulating rapamycin in PF127 micelles modulates cellula...

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