Results 1 to 10 of about 144,578 (239)
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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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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Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
Cells, 2023 Despite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on ...
S. Issa +3 more
semanticscholar +1 more source
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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Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer
BioDrugs, 2023 Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery vehicles resulting in three US Food and Drug Administration (FDA) and one European Medicines Agency (EMA)-approved AAV-based gene therapies.
Motahareh Arjomandnejad +3 more
semanticscholar +1 more source
Immunogenicity and toxicity of AAV gene therapy
Frontiers in Immunology, 2022 Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B.
H. Ertl
semanticscholar +1 more source
Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings
Frontiers in Medicine, 2022 Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to drive long-term transgene expression in gene therapy. While animal studies have shown promising results, the translatability of AAVs into clinical settings has ...
Hau Kiu Edna Au, M. Isalan, M. Mielcarek
semanticscholar +1 more source
Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?
Expert Opinion on Biological Therapy, 2022 While there has been substantial progress and notable achievements in the use of adeno-associated virus (AAV) gene therapy vectors for treatment of rare diseases, setbacks related to vector toxicity and immunogenicity represent major challenges for the ...
T. Kishimoto, R. Samulski
semanticscholar +1 more source
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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