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

CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease

利用 CRISPR-Cas9 技术编辑 HBG1 和 HBG2 启动子治疗镰状细胞病

Sharma, Akshay; Boelens, Jaap-Jan; Cancio, Maria; Hankins, Jane S; Bhad, Prafulla; Azizy, Marjohn; Lewandowski, Andrew; Zhao, Xiaojun; Chitnis, Shripad; Peddinti, Radhika; Zheng, Yan; Kapoor, Neena; Ciceri, Fabio; Maclachlan, Timothy; Yang, Yi; Liu, Yi; Yuan, Jianping; Naumann, Ulrike; Yu, Vionnie W C; Stevenson, Susan C; De Vita, Serena; LaBelle, James L

Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies

基因疗法非临床安全性评估的监管考量

Moffit, Jeffrey S; Blanset, Diann L; Lynch, Jessica L; MacLachlan, Timothy K; Meyer, Kathleen E; Ponce, Rafael; Whiteley, Laurence O

Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective

支持病毒载体递送基因疗法的非临床研究:欧洲制药工业协会基因治疗工作组的观点

Bolt, Michael W; Whiteley, Laurence O; Lynch, Jessica L; Lauritzen, Brian; Fernández de Henestrosa, Antonio R; MacLachlan, Timothy; Ulrich, Peter; Philip, Binu K; Mahalingaiah, Prathap Kumar; Fuller, Claudette L; Compton, David R

Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa

scAAV8-RLBP1治疗RLBP1视网膜色素变性的非临床安全性评价

MacLachlan, Timothy K; Milton, Mark N; Turner, Oliver; Tukov, Francis; Choi, Vivian W; Penraat, Jan; Delmotte, Marie-Hélène; Michaut, Lydia; Jaffee, Bruce D; Bigelow, Chad E

Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule.

在非人灵长类动物模型中,通过玻璃体内注射表达新型抗 VEGF 分子的 AAV2 载体抑制脉络膜新生血管形成

Lukason Michael, DuFresne Elizabeth, Rubin Hillard, Pechan Peter, Li Qiuhong, Kim Ivana, Kiss Szilard, Flaxel Christina, Collins Margaret, Miller Joan, Hauswirth William, Maclachlan Timothy, Wadsworth Samuel, Scaria Abraham

Insulin-like growth factor factor binding protein-2 is a novel mediator of p53 inhibition of insulin-like growth factor signaling

胰岛素样生长因子结合蛋白-2是p53抑制胰岛素样生长因子信号传导的一种新型介质

Grimberg, Adda; Coleman, Carrie M; Shi, Zonggao; Burns, Timothy F; MacLachlan, Timothy K; Wang, Wenge; El-Deiry, Wafik S