Results 21 to 30 of about 32,863 (259)
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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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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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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AAV gene therapy vectors in the TMJ
Clinical and Experimental Dental Research, 2022 AbstractObjectivesThe goal of this project was to evaluate the use of two adeno‐associated viral vector serotypes, adeno‐associated viral vectors (AAV)‐2 and AAV‐6, approved for and used for gene therapy in humans, for the delivery of therapeutic genes to the temporomandibular joint (TMJ) and the attendant sensory nerves.MethodsYoung adult wild‐type ...
Sabine M. Brouxhon +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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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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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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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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A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors
Frontiers in Immunology, 2023 IntroductionUse of adeno-associated virus (AAV) vectors is complicated by host immune responses that can limit transgene expression. Recent clinical trials using AAV vectors to deliver HIV broadly neutralizing antibodies (bNAbs) by intramuscular ...
Meredith E. Davis-Gardner +14 more
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Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
Molecular Therapy: Methods & Clinical Development, 2019 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.
Hildegard Büning, Arun Srivastava
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