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

Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns

delandistrogene moxeparvovec 基因疗法的真实世界结果:运动功能结果和新出现的安全性问题

Keselman, Dennis J; Small, Juliana C; Seneviratne, Tiffany; McCague, Sarah; Kaschak, Taylor L; Yum, Sabrina W; O'Brien, Andrea; Brandsema, John F; Diamond, Tamir; Loomes, Kathleen M; Lin, Kimberly Y; Wittlieb-Weber, Carol A; Mayer, Oscar H; Daniel, Scott G; Waldman, Amy T; Samelson-Jones, Benjamin J; Matesanz, Susan E; George, Lindsey A

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

利用患者特异性体内基因编辑技术治疗罕见遗传疾病

Musunuru, Kiran; Grandinette, Sarah A; Wang, Xiao; Hudson, Taylor R; Briseno, Kevin; Berry, Anne Marie; Hacker, Julia L; Hsu, Alvin; Silverstein, Rachel A; Hille, Logan T; Ogul, Aysel N; Robinson-Garvin, Nancy A; Small, Juliana C; McCague, Sarah; Burke, Samantha M; Wright, Christina M; Bick, Sarah; Indurthi, Venkata; Sharma, Shweta; Jepperson, Michael; Vakulskas, Christopher A; Collingwood, Michael; Keogh, Katie; Jacobi, Ashley; Sturgeon, Morgan; Brommel, Christian; Schmaljohn, Ellen; Kurgan, Gavin; Osborne, Thomas; Zhang, He; Kinney, Kyle; Rettig, Garrett; Barbosa, Christopher J; Semple, Sean C; Tam, Ying K; Lutz, Cathleen; George, Lindsey A; Kleinstiver, Benjamin P; Liu, David R; Ng, Kim; Kassim, Sadik H; Giannikopoulos, Petros; Alameh, Mohamad-Gabriel; Urnov, Fyodor D; Ahrens-Nicklas, Rebecca C

Use of CD19-targeted immune modulation to eradicate AAV-neutralizing antibodies.

利用 CD19 靶向免疫调节来根除 AAV 中和抗体

Doshi Bhavya S, Markmann Caroline A, Novak Noelle, Juarez Rojas Silvia, Davidson Robert, Chau Julia Q, Wang Wei, Carrig Sean, Martos Rus Cristina, Samelson-Jones Benjamin J, Small Juliana C, Bhoj Vijay G, George Lindsey A

Roctavian gene therapy for hemophilia A

罗克塔维亚基因疗法治疗血友病A

Samelson-Jones, Benjamin J; Small, Juliana C; George, Lindsey A

FVIII activity following FVIII protein infusion or FVIII gene transfer predicts the bleeding risk in hemophilia A rats

FVIII蛋白输注或FVIII基因转移后的FVIII活性可预测A型血友病大鼠的出血风险。

Lövgren, Karin M; Larsen, Malte S; Zintner, Shannon M; Small, Juliana C; Kjelgaard-Hansen, Mads; Häger, Mattias; Petersen, Maj; Wiinberg, Bo; Margaritis, Paris

Gene-based FVIIa prophylaxis modulates the spontaneous bleeding phenotype of hemophilia A rats

基于基因的FVIIa预防可以调节A型血友病大鼠的自发性出血表型

Zintner, Shannon M; Small, Juliana C; Pavani, Giulia; Dankner, Lynn; Marcos-Contreras, Oscar A; Gimotty, Phyllis A; Kjelgaard-Hansen, Mads; Wiinberg, Bo; Margaritis, Paris

Vaccine-induced T cells provide partial protection against high-dose rectal SIVmac239 challenge of rhesus macaques

疫苗诱导的T细胞可对恒河猴高剂量直肠SIVmac239攻击提供部分保护

Lasaro, Marcio O; Haut, Larissa H; Zhou, Xiangyang; Xiang, Zhiquan; Zhou, Dongming; Li, Yan; Giles-Davis, Wynetta; Li, Hua; Engram, Jessica C; Dimenna, Lauren J; Bian, Ang; Sazanovich, Marina; Parzych, Elizabeth M; Kurupati, Raj; Small, Juliana C; Wu, Te-Lang; Leskowitz, Rachel M; Klatt, Nicole R; Brenchley, Jason M; Garber, David A; Lewis, Mark; Ratcliffe, Sarah J; Betts, Michael R; Silvestri, Guido; Ertl, Hildegund C