日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression

预防性泼尼松龙通过 AAV5 辅助受体血小板衍生生长因子受体 α 上调和先天免疫抑制的新机制促进 AAV5 肝细胞转导

Britta Handyside, Lening Zhang, Bridget Yates, Lin Xie, Ashrafali Mohamed Ismail, Ryan Murphy, Brian Baridon, Cheng Su, Taren Bouwman, Linley Mangini, Jorden Tahquechi, Sandra Salcido, Wesley C Minto, William T Keenan, Ioanna Ntai, Choong-Ryoul Sihn, Sherry Bullens, Stuart Bunting, Sylvia Fong

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

腺相关病毒基因治疗血友病 A 后转基因 mRNA 和蛋白质产生的个体间差异

Sylvia Fong, Bridget Yates, Choong-Ryoul Sihn, Aras N Mattis, Nina Mitchell, Su Liu, Chris B Russell, Benjamin Kim, Adebayo Lawal, Savita Rangarajan, Will Lester, Stuart Bunting, Glenn F Pierce, K John Pasi, Wing Yen Wong

Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells

载体基因组丢失和表观遗传修饰导致哺乳动物和昆虫细胞中产生的 AAV5 载体转基因表达下降

Britta Handyside, Ashrafali Mohamed Ismail, Lening Zhang, Bridget Yates, Lin Xie, Choong-Ryoul Sihn, Ryan Murphy, Taren Bouwman, Chan Kyu Kim, Rolando De Angelis, Omair A Karim, Nicole L McIntosh, Michael Xavier Doss, Shilpa Shroff, Erno Pungor, Vikas S Bhat, Sherry Bullens, Stuart Bunting, Sylvia F

Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers

转导小鼠和非人类灵长类动物肝脏中 AAV5-hFVIII-SQ 载体基因组加工动力学的分子分析

Choong-Ryoul Sihn, Britta Handyside, Su Liu, Lening Zhang, Ryan Murphy, Bridget Yates, Lin Xie, Richard Torres, Chris B Russell, Charles A O'Neill, Erno Pungor, Stuart Bunting, Sylvia Fong

Intermittent enzyme replacement therapy with recombinant human β-galactosidase prevents neuraminidase 1 deficiency

重组人β-半乳糖苷酶间歇性酶替代疗法可预防神经氨酸酶 1 缺乏症

Amanda R Luu, Cara Wong, Vishal Agrawal, Nathan Wise, Britta Handyside, Melanie J Lo, Glenn Pacheco, Jessica B Felix, Alexander Giaramita, Alessandra d'Azzo, Jon Vincelette, Sherry Bullens, Stuart Bunting, Terri M Christianson, Charles M Hague, Jonathan H LeBowitz, Gouri Yogalingam

Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec

泼尼松龙不调节接受 AAV5-hFVIII-SQ 的小鼠中的因子 VIII 表达:Valoctocogene Roxaparvovec

Lening Zhang, Britta Handyside, Ryan Murphy, Choong-Ryoul Sihn, Lin Xie, Catherine Vitelli, Danielle Harmon, Sílvia Sisó, Su Liu, Sherry Bullens, Stuart Bunting, Sylvia Fong

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

BMN 270 基因治疗可使小鼠和灵长类动物的 FVIII 达到治疗水平,并使嗜血病小鼠的出血正常化

Stuart Bunting, Lening Zhang, Lin Xie, Sherry Bullens, Rajeev Mahimkar, Sylvia Fong, Krystal Sandza, Danielle Harmon, Bridget Yates, Britta Handyside, Choong-Ryoul Sihn, Nicole Galicia, Laurie Tsuruda, Charles A O'Neill, Anil Bagri, Peter Colosi, Shinong Long, Gordon Vehar, Barrie Carter

Targeting oxidized LDL improves insulin sensitivity and immune cell function in obese Rhesus macaques

针对氧化低密度脂蛋白可改善肥胖恒河猴的胰岛素敏感性和免疫细胞功能

Shijie Li, Paul Kievit, Anna-Karin Robertson, Ganesh Kolumam, Xiumin Li, Karin von Wachenfeldt, Christine Valfridsson, Sherry Bullens, Ilhem Messaoudi, Lindsay Bader, Kyra J Cowan, Amrita Kamath, Nicholas van Bruggen, Stuart Bunting, Björn Frendéus, Kevin L Grove

Pharmacokinetics and biodistribution of a human monoclonal antibody to oxidized LDL in cynomolgus monkey using PET imaging

使用 PET 成像研究人类抗氧化低密度脂蛋白单克隆抗体在猕猴体内的药代动力学和生物分布

Amrita V Kamath, Simon P Williams, Sherry Bullens, Kyra J Cowan, Yvonne Stenberg, Simon R Cherry, Stephen Rendig, David L Kukis, Chris Griesemer, Lisa A Damico-Beyer, Stuart Bunting

Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis

鞘内注射三肽基肽酶 1 可减少晚期婴儿神经元蜡样脂褐素沉积症犬模型中的溶酶体储存

Brian R Vuillemenot, Martin L Katz, Joan R Coates, Derek Kennedy, Pascale Tiger, Shinichi Kanazono, Peter Lobel, Istvan Sohar, Su Xu, Rhea Cahayag, Steve Keve, Eugen Koren, Stuart Bunting, Laurie S Tsuruda, Charles A O'Neill