Results 11 to 20 of about 73,809 (216)
Terminal glucose as a receptor for adeno-associated virus 44.9 [PDF]
Journal of VirologyThe isolation of novel adeno-associated virus (AAV) serotypes has led to significant advances in our understanding of parvovirus biology and vector development for gene therapy by identifying isolates with unique cell tropism and increased efficiency of ...
Giovanni Di Pasquale +4 more
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Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
Scientific Reports, 2022 Duchenne muscular dystrophy is a severe neuromuscular disease causing a progressive muscle wasting due to mutations in the DMD gene that lead to the absence of dystrophin protein.
Amédée Mollard +11 more
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Fluorescence Microscopy in Adeno-Associated Virus Research
Viruses, 2023 Research on adeno-associated virus (AAV) and its recombinant vectors as well as on fluorescence microscopy imaging is rapidly progressing driven by clinical applications and new technologies, respectively.
Susanne K. Golm +2 more
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Process Improvement of Adeno-Associated Virus Production
Frontiers in Chemical Engineering, 2022 Adeno-associated viruses (AAVs) have been well characterized and used to deliver therapeutic genes for diseases treatment in clinics and basic research. This study used the triple transient transfection of AAV-DJ/8 as a model expression system to develop
Jia-Shiung Guan +7 more
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Status and Prospect of Gene Therapy for Hemophilia
罕见病研究, 2022 Hemophilia is a congenital hemorrhagic disease caused by genetic abnormalities in coagulation factor Ⅷ or factor Ⅸ. Current conventional therapy to prevent bleeding requires frequent intravenous injections of coagulation factor concentrates from early ...
DAI Xinyue, ZHANG Lei
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Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease
Blood Advances, 2017 : Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction.
Randall K. Merling +14 more
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Molecular Therapy: Methods & Clinical Development, 2021 Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated
John W. Day +7 more
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Neuroscience Insights, 2019 Recombinant adeno-associated virus (rAAV) vectors have emerged as the safe vehicles of choice for long-term gene transfer in mammalian nervous system. Recombinant adeno-associated virus–mediated localized gene transfer in adult nervous system following ...
Asad Jan +4 more
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Familial and genetic overlap between Sjögren’s disease and other autoimmune diseases
Frontiers in ImmunologyPurposeTo evaluate familial clustering of Sjögren’s disease (SjD) with other autoimmune diseases and to characterize shared genetic architecture using an integrative genomic approach.MethodsMeta-analysis identified studies assessing autoimmune disease ...
Hitomi Ono-Minagi +6 more
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Virus Binding and Internalization Assay for Adeno-associated Virus
Bio-Protocol, 2017 The binding and internalization of adeno-associated virus (AAV) is an important determinant of viral infectivity and tropism. The ability to dissect these two tightly connected cellular processes would allow better understanding and provide insight on ...
Garrett Berry, Victor Tse
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