Search Results - "Grieger, Joshua C"

Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector by Grieger, Joshua C, Soltys, Stephen M, Samulski, Richard Jude

“…Adeno-associated virus (AAV) has shown great promise as a gene therapy vector in multiple aspects of preclinical and clinical applications. Many developments…”
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Manufacturing of recombinant adeno-associated viral vectors for clinical trials by Clément, Nathalie, Grieger, Joshua C

“…The ability to elicit robust and long-term transgene expression in vivo together with minimal immunogenicity and little to no toxicity are only a few features…”
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Adeno-associated virus vectorology, manufacturing, and clinical applications by Grieger, Joshua C, Samulski, R Jude

“…Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. The benefits of using AAV for gene therapy include long-term gene…”
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Production and characterization of adeno-associated viral vectors by Samulski, R Jude, Grieger, Joshua C, Choi, Vivian W

“…The adeno-associated virus (AAV) is one of the most promising viral vectors for human gene therapy. As with any potential therapeutic system, a thorough…”
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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector by Bowles, Dawn E, McPhee, Scott WJ, Li, Chengwen, Gray, Steven J, Samulski, Jade J, Camp, Angelique S, Li, Juan, Wang, Bing, Monahan, Paul E, Rabinowitz, Joseph E, Grieger, Joshua C, Govindasamy, Lakshmanan, Agbandje-McKenna, Mavis, Xiao, Xiao, Samulski, R Jude

“…Efficient and widespread gene transfer is required for successful treatment of Duchenne muscular dystrophy (DMD). Here, we performed the first clinical trial…”
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Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps by GRIEGER, Joshua C, SAMULSKI, Richard J

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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial by Monahan, Paul E, Sun, Junjiang, Gui, Tong, Hu, Genlin, Hannah, William B, Wichlan, David G, Wu, Zhijian, Grieger, Joshua C, Li, Chengwen, Suwanmanee, Thipparat, Stafford, Darrel W, Booth, Carmen J, Samulski, Jade J, Kafri, Tal, McPhee, Scott W J, Samulski, R Jude

“…Vector capsid dose-dependent inflammation of transduced liver has limited the ability of adeno-associated virus (AAV) factor IX (FIX) gene therapy vectors to…”
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Single Amino Acid Changes Can Influence Titer, Heparin Binding, and Tissue Tropism in Different Adeno-Associated Virus Serotypes by ZHIJIAN WU, ASOKAN, Aravind, GRIEGER, Joshua C, GOVINDASAMY, Lakshmanan, AGBANDJE-MCKENNA, Mavis, SAMULSKI, R. Jude

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Separate Basic Region Motifs within the Adeno-Associated Virus Capsid Proteins Are Essential for Infectivity and Assembly by GRIEGER, Joshua C, SNOWDY, Stephen, SAMULSKI, Richard J

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Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium by Li, Wuping, Zhang, Liqun, Johnson, Jarrod S, Zhijian, Wu, Grieger, Joshua C, Ping-Jie, Xiao, Drouin, Lauren M, Agbandje-McKenna, Mavis, Pickles, Raymond J, Samulski, R Jude

“…Recombinant adeno-associated virus (AAV) vectors expressing the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been used to deliver CFTR…”
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Gene therapy approaches for equine osteoarthritis by Thampi, Parvathy, Samulski, R Jude, Grieger, Joshua C, Phillips, Jennifer N, McIlwraith, C Wayne, Goodrich, Laurie R

“…With an intrinsically low ability for self-repair, articular cartilage injuries often progress to cartilage loss and joint degeneration resulting in…”
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Corrigendum: Gene therapy approaches for equine osteoarthritis by Thampi, Parvathy, Samulski, R Jude, Grieger, Joshua C, Phillips, Jennifer N, McIlwraith, C Wayne, Goodrich, Laurie R

“…[This corrects the article DOI: 10.3389/fvets.2022.962898.]…”
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Surface-Exposed Adeno-Associated Virus Vp1-NLS Capsid Fusion Protein Rescues Infectivity of Noninfectious Wild-Type Vp2/Vp3 and Vp3-Only Capsids but Not That of Fivefold Pore Mutant Virions by Grieger, Joshua C, Johnson, Jarrod S, Gurda-Whitaker, Brittney, Agbandje-McKenna, Mavis, Samulski, R Jude

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Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in a mono-iodoacetate-induced osteoarthritis rat model by Wang, Gensheng, Evans, Christopher H, Benson, Janet M, Hutt, Julie A, Seagrave, JeanClare, Wilder, Julie A, Grieger, Joshua C, Samulski, R Jude, Terse, Pramod S

“…Interleukin-1 (IL-1) plays an important role in the pathophysiology of osteoarthritis (OA), and gene transfer of IL-1 receptor antagonist (IL-1Ra) holds…”
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Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis by Goodrich, Laurie R, Phillips, Jennifer N, McIlwraith, C Wayne, Foti, Stacey B, Grieger, Joshua C, Gray, Steven J, Samulski, R Jude

“…Osteoarthritis (OA) affects over 40 million people annually. We evaluated interleukin-1 receptor antagonist (IL-1ra) gene transfer in an equine model based on…”
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Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors by Kim, Ah Young, Duerr, Felix Michael, Phillips, Jennifer N., Samulski, Richard Jude, Grieger, Joshua C., Goodrich, Laurie R.

“…A formal screening of self-complementary adeno-associated virus (scAAV) vector serotypes in canine joint tissues has not been performed to date. Selecting…”
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Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications by Grieger, Joshua C, Samulski, Richard J

“…Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. AAV vectors have successfully been utilized to promote sustained gene…”
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