Results 41 to 50 of about 9,435,635 (153)
Adeno-Associated Virus VP1u Exhibits Protease Activity
Viruses, 2019 Adeno-associated viruses (AAVs) are being developed for gene delivery applications, with more than 100 ongoing clinical trials aimed at the treatment of monogenic diseases.
Justin J. Kurian +7 more
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, 2023 Adeno-‐associated virus (AAV) is a leading candidate as a gene therapy vector, but as with other vectors, one of the limitations is a host-neutralizing immune ...
McCraw, Dustin
core +1 more source
Pharmacology of Recombinant Adeno-associated Virus Production
Molecular Therapy: Methods & Clinical Development, 2018 Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases.
Magalie Penaud-Budloo +3 more
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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]
, 2002 The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Hanawa, H +12 more
core
VirusesToday, adeno-associated virus (AAV)-based vectors are arguably the most promising in vivo gene delivery vehicles for durable therapeutic gene expression. Advances in molecular engineering, high-throughput screening platforms, and computational techniques
Estrella Lopez-Gordo +4 more
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Journal of Integrative Neuroscience, 2019 The effects of cellular prion protein on rapid eye movement sleep deprivation-induced spatial memory impairment were investigated, and the related mechanisms explored.
Li Hu +5 more
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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
, 2001 Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Hanawa, H +5 more
core
AAV-based vectors for human diseases modeling in laboratory animals
Frontiers in MedicineThe development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models.
Timur I. Aliev, Dmitry V. Yudkin
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, 2022 Adeno-associated virus (AAV)-mediated gene transfer is an efficient method of gene over-expression in the vestibular end organs. However, AAV has limited usefulness for delivering a large gene, or multiple genes, due to its small packaging capacity ...
Jing-Ying Guo (743732) +9 more
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Potential for cellular stress response to hepatic factor VIII expression from AAV vector
Molecular Therapy: Methods & Clinical Development, 2016 Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin +5 more
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