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The Menace of Severe Adverse Events and Deaths Associated with Viral Gene Therapy and Its Potential Solution by Artyom Kachanov; Anastasiya Kostyusheva; Sergey Brezgin; Ivan Karandashov; Natalia Ponomareva; Andrey Tikhonov; Alexander Lukashev; Vadim Pokrovsky; Andrey A. Zamyatnin; Alessandro Parodi; Vladimir Chulanov; Dmitry Kostyushev is a Biochemistry, Genetics and Molecular Biology article available to read on EtoBox.
What is The Menace of Severe Adverse Events and Deaths Associated with Viral Gene Therapy and Its Potential Solution about?
## Abstract Over the past decade, in vivo gene replacement therapy has significantly advanced, resulting in market approval of numerous therapeutics predominantly relying on adeno‐associated viral vectors (AAV). While viral vectors have undeniably addressed several critical healthcare challenges, their clinical application has unveiled a range of limitations and safety concerns. This review highlights the emerging challenges in the field of gene therapy. At first, we discuss both the role of biological barriers in viral gene therapy with a focus on AAVs, and review current landscape of in vivo human gene therapy. We delineate advantages and disadvantages of AAVs as gene delivery vehicles, mostly from the safety perspective (hepatotoxicity, cardiotoxicity, neurotoxicity, inflammatory responses etc.), and outline the mechanisms of adverse events in response to AAV. Contribution of every aspect of AAV vectors (genomic structure, capsid proteins) and host responses to injected AAV is considered and substantiated by basic, translational and clinical studies. The updated evaluation of recent AAV clinical trials and current medical experience clearly shows the risks of AAVs that sometimes
Who reads The Menace of Severe Adverse Events and Deaths Associated with Viral Gene Therapy and Its Potential Solution?
It is typically read by researchers, students, and practitioners in Biochemistry, Genetics and Molecular Biology.
- Author
- Artyom Kachanov; Anastasiya Kostyusheva; Sergey Brezgin; Ivan Karandashov; Natalia Ponomareva; Andrey Tikhonov; Alexander Lukashev; Vadim Pokrovsky; Andrey A. Zamyatnin; Alessandro Parodi; Vladimir Chulanov; Dmitry Kostyushev
- Publisher
- Wiley
- Published
- 2024
- Language
- EN
- Field
- Biochemistry, Genetics and Molecular Biology (Life Sciences)