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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 Calcedo +2 more
exaly +6 more sources
Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions
Frontiers in Immunology, 2021 Adeno-associated virus (AAV) vector-based gene therapy is currently the only in vivo gene therapy approved in the US and Europe. The recent tragic death of three children in a clinical trial to treat X-Linked Myotubular Myopathy by delivering ...
Thomas Weber
exaly +3 more sources
AAV: An Overview of Unanswered Questions [PDF]
Human Gene Therapy, 2017 AAV has been studied for 55 years and has been developed as a vector for about 35 years. By now, there is a fairly good idea of the dimensions of what would be useful to know to employ AAV optimally as a vector, but there are still many unanswered questions within the system. As with all biological systems, each good experiment raises further questions
Kenneth I Berns, Nicholas Muzyczka
exaly +3 more sources
The effect of various forms of treatment of vasculitis on C3, C4 and C5a complement levels in infants and children attending Assiut University Children Hospital (AUCH) [PDF]
E3S Web of Conferences, 2023 Vasculitis is an umbrella term for various and heterogeneous disorders sharing the presence of inflammation of blood vessel walls (Geetha & Jefferson, 2020).
Fatthi Dalal Abdullah
doaj +1 more source
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
openaire +3 more sources
Frontiers in Immunology, 2021 PurposeTo develop a novel method to quantify the amount of fibrosis in the salivary gland and to investigate the relationship between fibrosis and specific symptoms associated with Sjögren’s syndrome (SS) using this method.Materials and MethodsParaffin ...
Hongen Yin +6 more
doaj +1 more source
Neutrophils in ANCA-associated vasculitis: Mechanisms and implications for management
Frontiers in Pharmacology, 2022 Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of systemic autoimmune diseases, which is typified by inflammatory necrosis predominantly affecting the small vessels and often accompanied by positive ANCA.
Shangqing Ge +16 more
doaj +1 more source
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
openaire +4 more sources
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
doaj +1 more source
Brain Sciences, 2020 Recently, adeno-associated virus (AAV)-mediated gene therapies have attracted clinical interest for treating neurodegenerative diseases including spinal muscular atrophy (SMA), Canavan disease (CD), Parkinson’s disease (PD), and Friedreich’s ...
Barbara A. Perez +4 more
doaj +1 more source