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

Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood

接受成人剂量 AAV5-hFVIII-SQ 治疗的幼鼠在成年后仍能表达治疗性因子 VIII

Lening Zhang, Bridget Yates, Ryan Murphy, Su Liu, Lin Xie, Britta Handyside, Choong-Ryoul Sihn, Taren Bouwman, Nicole Galicia, Danielle Tan, Carlos Fonck, Jeremy Arens, Annie Clark, Weiming Zhang, Sundeep Chandra, Jaydeep Srimani, Jennifer Holcomb, Andrea Van Tuyl, Joshua Henshaw, Christian Vetterma

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

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

Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Sanfilippo B 型小鼠脑室内注射 BMN 250 清除硫酸肝素并减轻中枢神经系统病理

Mika Aoyagi-Scharber, Danielle Crippen-Harmon, Roger Lawrence, Jon Vincelette, Gouri Yogalingam, Heather Prill, Bryan K Yip, Brian Baridon, Catherine Vitelli, Amanda Lee, Olivia Gorostiza, Evan G Adintori, Wesley C Minto, Jeremy L Van Vleet, Bridget Yates, Sara Rigney, Terri M Christianson, Pascale