Results 1 to 10 of about 131,610 (332)
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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PEP-TP56 enhances AAV transduction in a serotype-independent manner
HeliyonAdeno-associated viruses (AAVs) have emerged as the most prominent gene therapy vectors. However, the need for high AAV doses to achieve clinical efficacy remains a key challenge to AAV utility in the clinic.
Olaniyi Olarewaju +4 more
doaj +2 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
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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
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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
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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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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, Thomas Weber
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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
core +6 more sources
Frontiers in Molecular Biosciences, 2023 Huntington’s disease (HD) is a multi-tissue failure disorder for which there is no cure. We have previously shown an effective therapeutic approach limited mainly to the central nervous system, based on a synthetic zinc finger (ZF) transcription ...
Michal Mielcarek +3 more
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