Year: 2019
Type: review
Abstract: In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical trials and their efficacy in at least eight human diseases are now firmly documented. Despite these remarkable achievements, it has also become abundantly clear that the full potential of first generation AAV vectors composed of naturally occurring capsids is not likely to be realized, since the wild-type AAV did not evolve for the purpose of therapeutic gene delivery. In this article, we provide a brief historical account of the progress that has been made in the development of capsid-modified , next-generation AAV vectors to ensure both the safety and efficacy of these vectors in targeting a wide variety of human diseases.
Authors: Hildegard Büning, Arun Srivastava
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,500
Funders: Kitzman Foundation, Children's Miracle Network Hospitals, Deutsche Forschungsgemeinschaft, Bundesministerium für Bildung und Forschung, Ministerium für Wissenschaft, Forschung und Kunst Baden-Württemberg, Niedersächsisches Ministerium für Wissenschaft und Kultur, National Institutes of Health, National Heart, Lung, and Blood Institute, National Institute of General Medical Sciences, National Institute of Allergy and Infectious Diseases, National Institute of Biomedical Imaging and Bioengineering
Awards: AAV2 and hepatocellular carcinoma, Safety and efficacy of optimized AAV3 vectors in murine and NHP livers, Development of Optimized AAV Serotype Vectors for Human Gene Therapy, G4253744469, Next Generation of Recombinant AAV Serotype For Gene Therapy, Mechanism of high-efficiency transduction of hepatocytes by optimized AAV vectors
OpenAlex ID: W2912684492