Results 11 to 20 of about 70,372 (263)
Adeno‐associated virus (AAV)-based gene therapy for glioblastoma
Cancer Cell International, 2021 Glioblastoma (GBM) is the most common and malignant Grade IV primary craniocerebral tumor caused by glial cell carcinogenesis with an extremely poor median survival of 12–18 months.
Xin Xu +9 more
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Transduction of Salivary Gland Acinar Cells with a Novel AAV Vector 44.9
Molecular Therapy: Methods & Clinical Development, 2020 The loss of salivary gland function caused by radiation therapy of the head and neck or autoimmune disease such as Sjögren’s syndrome is a serious condition that affects a patient’s quality of life. Due to the combined exocrine and endocrine functions of
Giovanni Di Pasquale +14 more
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Advances in Rheumatology, 2022 Background Sjögren Syndrome (SS) is a systemic autoimmune disease with a wide spectrum of manifestations that can lead to misdiagnosis. This study describes and compares demographic, clinical, serological, and histopathological data from subjects with SS
Fabíola Reis de Oliveira +6 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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Adeno-associated Virus DNA Replication [PDF]
Cold Spring Harbor Symposia on Quantitative Biology, 1979 ABSTRACT. A model for the replication of adeno-associated virus (AAV) DNA is presented. The AAV genome is a linear single-stranded DNA molecule which contains a 145 nucleotide inverted terminal repetition of which the first 125 nucleotides form a palindromic sequence.
K I, Berns +3 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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