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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
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
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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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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
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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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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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The HLA class I immunopeptidomes of AAV capsid proteins
Frontiers in Immunology, 2023 IntroductionCellular immune responses against AAV vector capsid represent an obstacle for successful gene therapy. Previous studies have used overlapping peptides spanning the entire capsid sequence to identify T cell epitopes recognized by AAV-specific ...
Carlos A. Brito-Sierra +3 more
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Plectin-1 targeted AAV vector for the molecular imaging of pancreatic cancer
Frontiers in Oncology, 2013 Pancreatic ductal adenocarcinoma (PDAC) is highly malignant disease that is the 4th leading cause of cancer-related death in the US. Gene therapy using AAV vectors to selectively deliver genes to PDAC cells is an attractive treatment option for ...
Prasad R. Konkalmatt +6 more
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