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

Addendum to: Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases

附录:利用小型高活性和特异性工程化RNA引导核酸酶改进CRISPR基因组编辑

Schmidt, Moritz J; Gupta, Ashish; Bednarski, Christien; Gehrig-Giannini, Stefanie; Richter, Florian; Pitzler, Christian; Gamalinda, Michael; Galonska, Christina; Takeuchi, Ryo; Wang, Kui; Reiss, Caroline; Dehne, Kerstin; Lukason, Michael J; Noma, Akiko; Park-Windhol, Cindy; Allocca, Mariacarmela; Kantardzhieva, Albena; Sane, Shailendra; Kosakowska, Karolina; Cafferty, Brian; Tebbe, Jan; Spencer, Sarah J; Munzer, Scott; Cheng, Christopher J; Scaria, Abraham; Scharenberg, Andrew M; Cohnen, André; Coco, Wayne M

Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

支持治疗GUCY2D相关Leber先天性黑蒙症的I/II期临床试验的临床前研究

Boye, Sanford L; O'Riordan, Catherine; Morris, James; Lukason, Michael; Compton, David; Baek, Rena; Elmore, Dana M; Peterson, James J; Fajardo, Diego; McCullough, K Tyler; Scaria, Abraham; McVie-Wylie, Alison; Boye, Shannon E

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

不同血清型重组AAV载体的通用纯化方法

Nass, Shelley A; Mattingly, Maryellen A; Woodcock, Denise A; Burnham, Brenda L; Ardinger, Jeffrey A; Osmond, Shayla E; Frederick, Amy M; Scaria, Abraham; Cheng, Seng H; O'Riordan, Catherine R

Gene Therapies for Neovascular Age-Related Macular Degeneration

基因疗法治疗新生血管性年龄相关性黄斑变性

Pechan, Peter; Wadsworth, Samuel; Scaria, Abraham

Aurintricarboxylic acid increases yield of HSV-1 vectors

金精三羧酸可提高HSV-1载体的产量

Pechan, Peter; Ardinger, Jeffery; Ketavarapu, Jyothi; Rubin, Hillard; Wadsworth, Samuel C; Scaria, Abraham

AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice

AAV5介导的sFLT01基因疗法可阻止Ccl2(-/-)/Cx3cr1(-/-)小鼠的视网膜病变。

Tuo, Jingsheng; Pang, Ji-Jing; Cao, Xiaoguang; Shen, Defen; Zhang, Jun; Scaria, Abraham; Wadsworth, Samuel C; Pechan, Peter; Boye, Sanford L; Hauswirth, William W; Chan, Chi-Chao

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

Cytotoxic T lymphocyte responses to transgene product, not adeno-associated viral capsid protein, limit transgene expression in mice

小鼠体内转基因表达受限于针对转基因产物的细胞毒性T淋巴细胞反应,而非腺相关病毒衣壳蛋白。

Siders, William M; Shields, Jacqueline; Kaplan, Johanne; Lukason, Michael; Woodworth, Lisa; Wadsworth, Sam; Scaria, Abraham

Mutations within the ADP (E3-11.6K) protein alter processing and localization of ADP and the kinetics of cell lysis of adenovirus-infected cells

ADP (E3-11.6K) 蛋白的突变会改变 ADP 的加工和定位,以及腺病毒感染细胞的裂解动力学。

Tollefson, Ann E; Scaria, Abraham; Ying, Baoling; Wold, William S M