Results 21 to 30 of about 326,770 (247)
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
openaire +3 more sources
BMC Research Notes, 2017 Objective Recombinant adeno-associated virus (AAV)-based vectors are characterized by their robust and safe transgene delivery. The CRISPR/Cas9 and guide RNA (gRNA) system present a promising genome-editing platform, and a recent development of a shorter
Salma G. Morsy +8 more
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AAV- based vector improvements unrelated to capsid protein modification
Frontiers in Medicine, 2023 Recombinant adeno-associated virus (rAAV) is the leading platform for delivering genetic constructs in vivo. To date, three AAV-based gene therapeutic agents have been approved by the FDA and are used in clinical practice. Despite the distinct advantages
Ekaterina M. Shitik +2 more
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A comparative analysis of constitutive promoters located in adeno-associated viral vectors.
PLoS ONE, 2014 The properties of constitutive promoters within adeno-associated viral (AAV) vectors have not yet been fully characterized. In this study, AAV vectors, in which enhanced GFP expression was directed by one of the six constitutive promoters (human β-actin,
Lkhagvasuren Damdindorj +6 more
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Frontiers in Pharmacology, 2022 Adeno-associated virus (AAV) mediated gene therapy has been successfully applied in clinical trials, including hemophilia. Novel AAV vectors have been developed with enhanced transduction and specific tissue tropism.
Wenwei Shao +12 more
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Efficient AAV-mediated Gene Targeting Using 2A-based Promoter-trap System
Bio-Protocol, 2016 Adeno-associated virus (AAV)-based targeting vectors have 1-4-log higher gene targeting efficiencies compared with plasmid-based targeting vectors. The efficiency of AAV-mediated gene targeting is further increased by introducing a promoter-trap system ...
Sivasundaram Karnan +6 more
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Triple Vectors Expand AAV Transfer Capacity in the Retina [PDF]
, 2017 Retinal gene transfer with adeno-associated viral (AAV) vectors holds great promise for the treatment of inherited retinal degenerations (IRDs). One limit of AAV is its transfer capacity of about 5 kb, which can be expanded to about 9 kb, using dual AAV ...
Francesca Simonelli +22 more
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PLoS ONE, 2019 The engineered AAV-PHP.B family of adeno-associated virus efficiently delivers genes throughout the mouse central nervous system. To guide their application across disease models, and to inspire the development of translational gene therapy vectors for ...
Qin Huang +10 more
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Generation of enhanced gene delivery vectors by directed evolution of adeno-associated virus [PDF]
, 2006 Despite promising advance in the development of viral vectors based on AAV for human gene therapy, several major hurdles for a more general use remain.
Endell, Jan
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Humoral immune response to AAV
Frontiers in Immunology, 2013 Adeno-associated virus (AAV) is a member of the family parvoviridae that has been widely used as a vector for gene therapy because of its safety profile, its ability to transduce both dividing and non-dividing cells, and its low immunogenicity.
Roberto eCalcedo, James M Wilson
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