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

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

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy

预先存在的免疫力和灵长类动物体内低表达水平凸显了肝脏靶向AAV8介导的基因治疗在转化应用方面面临的挑战。

Hurlbut, Gregory D; Ziegler, Robin J; Nietupski, Jennifer B; Foley, Joseph W; Woodworth, Lisa A; Meyers, Elizabeth; Bercury, Scott D; Pande, Nilesh N; Souza, David W; Bree, Mark P; Lukason, Michael J; Marshall, John; Cheng, Seng H; Scheule, Ronald K

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