Results 31 to 40 of about 116,785 (352)
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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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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Profiling of Glycan Receptors for Minute Virus of Mice in Permissive Cell Lines Towards Understanding the Mechanism of Cell Recognition [PDF]
, 2013 The recognition of sialic acids by two strains of minute virus of mice (MVM), MVMp (prototype) and MVMi (immunosuppressive), is an essential requirement for successful infection.
Agbandje-McKenna, M +12 more
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In vivo genetic manipulation of inner ear connexin expression by bovine adeno-Associated viral vectors [PDF]
, 2017 We have previously shown that in vitro transduction with bovine adeno-associated viral (BAAV) vectors restores connexin expression and rescues gap junction coupling in cochlear organotypic cultures from connexin-deficient mice that are models DFNB1 ...
Campioni, Matteo +7 more
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Adeno-associated virus: fit to serve [PDF]
Current Opinion in Virology, 2014 Adeno-associated virus (AAV) is a helper-dependent parvovirus which has not been linked with human disease. This aspect, in combination with its broad cell and tissue tropism, and limited viral host response has made it an attractive vector system for gene therapy.
Eric Zinn, Luk H Vandenberghe
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Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]
, 2013 Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S +8 more
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Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy [PDF]
, 2016 Acknowledgments Blood biochemistry analysis and serum analysis were performed by the Easter Bush Pathology Department, University of Edinburgh. Animal husbandry was performed by Centre for Integrative Physiology bio-research restructure technical staff ...
Azzouz, Mimoun +15 more
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Adeno-associated virus integration: virus versus vector [PDF]
Gene Therapy, 2008 Although a large percentage of the world population is seropositive for exposure to various strains of adeno-associated virus (AAV), a human parvovirus, AAV has never been identified as an etiologic agent of human disease. Most likely contributing to the pronounced lack of pathogenicity is the fact that AAV is a naturally defective virus that requires ...
openaire +2 more sources
The natural dietary genistein boosts bacteriophage-mediated cancer cell killing by improving phage-targeted tumor cell transduction [PDF]
, 2016 Gene therapy has long been regarded as a promising treatment for cancer. However, cancer gene therapy is still facing the challenge of targeting gene delivery vectors specifically to tumors when administered via clinically acceptable non-invasive ...
Albahrani, M +5 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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