Results 11 to 20 of about 32,863 (259)
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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AAV Vectors for the Nucleolus [PDF]
Molecular Therapy, 2012 During the replication of wild-type adeno-associated virus (AAV), viral capsids are assembled in the nucleolus,1 and the capsid proteins interact with the nucleolar proteins nucleolin (NCL) and nucleophosmin (NPM1), possibly promoting their transport from the cytoplasm into the nucleolus.2,3 In the case of replication-incompetent AAV vectors, the ...
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Molecular Therapy: Methods & Clinical Development, 2020 The recombinant adeno-associated virus (AAV) vector is one of the most utilized viral vectors in gene therapy due to its robust, long-term in vivo transgene expression and low toxicity.
James H. Kurasawa +9 more
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Microorganisms, 2021 Nanobodies are camelid-derived single-domain antibodies that present some advantages versus conventional antibodies, such as a smaller size, and higher tissue penetrability, stability, and hydrophilicity. Although nanobodies can be delivered as proteins,
Noelia Silva-Pilipich +2 more
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AAV Vectored Immunoprophylaxis for Filovirus Infections [PDF]
Tropical Medicine and Infectious Disease, 2020 Filoviruses are among the deadliest infectious agents known to man, causing severe hemorrhagic fever, with up to 90% fatality rates. The 2014 Ebola outbreak in West Africa resulted in over 28,000 infections, demonstrating the large-scale human health and economic impact generated by filoviruses.
Amira D. Rghei +8 more
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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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