Results 11 to 20 of about 144,727 (384)
Humoral immune response to AAV [PDF]
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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T Cell-Mediated Immune Responses to AAV and AAV Vectors [PDF]
Frontiers in Immunology, 2021 Adeno-associated virus (AAV)-mediated gene transfer has benefited patients with inherited diseases, such as hemophilia B, by achieving long-term expression of the therapeutic transgene.
H. Ertl
semanticscholar +5 more sources
AAV genome modification for efficient AAV production
Heliyon, 2023 The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases.
Walaa Asaad +6 more
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Blood, 2021 In this issue of Blood, Konkle et al report that 7 of 8 participants in a phase 1/2 trial of adeno-associated virus (AAV) vector (BAX335) for factor IX (FIX)-Padua gene transfer in patients with hemophilia B did not maintain expression despite steroid intervention, which the authors hypothesize is a result of the innate immune stimulatory effect of ...
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AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6 [PDF]
Molecular Therapy - Methods & Clinical Development, 2018 Under intravenous delivery, recombinant adeno-associated vectors (rAAVs) interact with blood-borne components in ways that can critically alter their therapeutic efficiencies. We have previously shown that interaction with human galectin 3 binding protein dramatically reduces rAAV-6 efficacy, whereas binding of mouse C-reactive protein improves rAAV-1 ...
Denard, Jérôme +9 more
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Frontiers in Genome Editing, 2023 Gene therapy is a fast developing field of medicine with hundreds of ongoing early-stage clinical trials and numerous preclinical studies. Genome editing (GE) now is an increasingly important technology for achieving stable therapeutic effect in gene ...
Alena Shakirova +4 more
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Characterizing infection in anti-neutrophil cytoplasmic antibody-associated vasculitis:results from a longitudinal, matched-cohort data linkage study [PDF]
, 2020 Objectives: Infection exerts a major burden in ANCA-associated vasculitis (AAV), however, its precise extent and nature remains unclear. In this national study we aimed to longitudinally quantify, characterize and contextualize infection risk in AAV ...
Amft, Nicole +11 more
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Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines
Frontiers in Cell and Developmental Biology, 2023 Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults and affects mainly the skeletal muscle, heart, and brain. DM1 is caused by a CTG repeat expansion in the 3′UTR region of the DMPK gene that sequesters muscleblind ...
Camila F. Almeida +16 more
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Molecular Therapy, 2015 The paper recently published in Nature by Gardner et al.1 marks yet another milestone in the development of adeno-associated virus (AAV) as an effective gene delivery vehicle, and is an appropriate way to highlight the fiftieth anniversary of the discovery of AAV. Using simple intramuscular injections in primates, Gardner et al.
Kenneth I. Berns, Nicholas Muzyczka
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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice
Frontiers in Molecular Neuroscience, 2021 Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder characterized by accumulation of sulfatides in both glial cells and neurons. MLD results from an inherited deficiency of arylsulfatase A (ARSA) and myelin degeneration in the central and ...
Emilie Audouard +6 more
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