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

Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A

重度血友病A患者血液中valoctocogene roxaparvovec的生物分布和载体脱落

Agarwal, Suresh; Sandza, Krystal; Obrochta Moss, Kristin; Vora, Monica; Bowen, Alisa; Bunch, Brenna; Holcomb, Jennifer; Robinson, Tara M; Jayaram, Kala; Russell, Chris B; Zoog, Stephen; Vettermann, Christian; Henshaw, Joshua

Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer

利用基于免疫捕获的qPCR技术,在VIII因子基因转移后进行超灵敏的AAV衣壳检测

Sandza, Krystal; Clark, Annie; Koziol, Elli; Akeefe, Hassibullah; Yang, Fan; Holcomb, Jennifer; Patton, Kathryn; Hammon, Kevin; Mitchell, Nina; Wong, Wing Y; Zoog, Stephen J; Kim, Benjamin; Henshaw, Joshua; Vettermann, Christian

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

既有免疫对基于AAV5基因治疗的非临床药效学的影响

Long, Brian R; Sandza, Krystal; Holcomb, Jennifer; Crockett, Lucy; Hayes, Gregory M; Arens, Jeremy; Fonck, Carlos; Tsuruda, Laurie S; Schweighardt, Becky; O'Neill, Charles A; Zoog, Stephen; Vettermann, Christian

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

Strategy to detect pre-existing immunity to AAV gene therapy

检测针对AAV基因疗法的预存免疫力的策略

Falese, L; Sandza, K; Yates, B; Triffault, S; Gangar, S; Long, B; Tsuruda, L; Carter, B; Vettermann, C; Zoog, S J; Fong, S