Results 1 to 10 of about 1,183 (67)
Molecular Therapy: Nucleic Acids, 2023 Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in 332 phase I/II/III clinical trials in a number of human diseases, and in some cases, remarkable clinical efficacy has also been achieved.
Arun Srivastava
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Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Molecular Therapy: Methods & Clinical Development, 2018 Pre-existing immunity to adeno-associated virus (AAV) is highly prevalent in humans and can profoundly impact transduction efficiency. Despite the relevance to AAV-mediated gene transfer, relatively little is known about the fate of AAV vectors in the ...
Zachary Fitzpatrick +12 more
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Biomedicines, 2014 Adeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm ...
J. Fraser Wright
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Co-transduction of dual-adeno-associated virus vectors in the neonatal and adult mouse utricles
Frontiers in Molecular Neuroscience, 2022 Adeno-associated virus (AAV)-mediated gene transfer is an efficient method of gene over-expression in the vestibular end organs. However, AAV has limited usefulness for delivering a large gene, or multiple genes, due to its small packaging capacity (<
Zhong-Rui Chen +19 more
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Hepatology Communications, 2023 Background and Aims:. Adeno-associated virus (AAV) vectors are widely used to deliver therapeutic transgenes to distinct tissues, including the liver.
Daniel Stone +20 more
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Frontiers in Immunology, 2022 One of the major goals of in vivo gene transfer is to achieve long-term expression of therapeutic transgenes in terminally differentiated cells. The extensive clinical experience and the recent approval of Luxturna® (Spark Therapeutics, now Roche) and ...
David-Alexandre Gross +7 more
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Engineering a highly durable adeno-associated virus receptor for analytical applications
Molecular Therapy: Methods & Clinical Development, 2023 Adeno-associated virus (AAV) is a major viral vector used in gene therapy. There are multiple AAV serotypes, and many engineered AAV serotypes are developed to alter their tissue tropisms with capsid modification.
Kouhei Yoshida +8 more
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Planet of the AAVs: The Spinal Cord Injury Episode
Biomedicines, 2021 The spinal cord injury (SCI) is a medical and life-disrupting condition with devastating consequences for the physical, social, and professional welfare of patients, and there is no adequate treatment for it.
Katerina Stepankova +2 more
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Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studies
Frontiers in Medicine, 2023 Retinal degenerative diseases, including glaucoma, age-related macular degeneration, diabetic retinopathy, and a broad range of inherited retinal diseases, are leading causes of irreversible vision loss and blindness. Gene therapy is a promising and fast-
Xue Xia, Xinzheng Guo
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Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
Molecular Therapy: Methods & Clinical Development, 2017 Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production.
Qizhao Wang +10 more
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