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

A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina

一种用于开发靶向小鼠视网膜的基因组编辑蛋白递送载体的组合合成策略

Zhang, Jianye; Hołubowicz, Rafał; Smidak, Roman; Hu, Yulun; Du, Samuel W; Felgner, Jiin H; Palczewska, Grazyna; Rodrigues Menezes, Carolline; Risaliti, Eleonora; Dong, Zhiqian; Ma, Xiuli; Shayegan, Mojtaba H; Chen, Paul Z; Xing, Li; Hołubowicz, Maria; Li, Bowen; Liu, David R; Felgner, Philip L; Tochtrop, Gregory P; Palczewski, Krzysztof

Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform

FDA新的合理机制框架对开发个性化体内先导基因编辑平台的影响

Feierman, Emily R; Whittaker, Madelynn N; Quigley, Aidan; Brooks, Dominique L; McVeigh, Paige; Nan, Angela X; Hsu, Alvin; Said, Hooda; Soliman, Ousamah Younoss; Giovenco, Ryan; Davidson, Beverly L; Alameh, Mohamad-Gabriel; Liu, David R; Wang, Xiao; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C

Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.

在小鼠模型中对 CRISPR-Cas9、慢病毒转导和碱基编辑治疗镰状细胞病进行比较分析。

Butt Henna, Sathish Shruti, London Evan, Le Anh, Li Quan, Gudmundsdottir Bjorg, Lee Duck-Yeon, Burke Emma V, Yates Bradley P, Liu David R, Hsieh Matthew, Leonard Alexis, Eaton William A, Uchida Naoya, Pierciey Francis J Jr, Newby Gregory A, Tisdale John F, Demirci Selami

Mechanistic machine learning enables interpretable and generalizable prediction of prime editing outcomes

机制机器学习能够对主要编辑结果进行可解释且可推广的预测。

Hsu, Alvin; Chen, Peter J; Li, Angus H; Hemez, Colin F; Gao, Xin D; Terrey, Markus; Nelson, Charlie; Selvam, Vijay; Cristian, Ana; McElroy, Amber N; Steinbeck, Benjamin J; Mahadeshwar, Gandhar K; Pandey, Smriti; Barsdale, Zachary; Chen, Paul Z; Sousa, Alexander A; Sakai, Holt A; Silverstein, Rachel A; Morad, Ilias; Krueger, Ryan K; Shen, Max W; Kleinstiver, Benjamin P; Lutz, Cathleen M; Tolar, Jakub; Blazar, Bruce R; Osborn, Mark J; Liu, David R

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

Author Correction: In vivo base editing extends lifespan of a humanized mouse model of prion disease

作者更正:体内碱基编辑延长了朊病毒病人源化小鼠模型的寿命

An, Meirui; Davis, Jessie R; Levy, Jonathan M; Serack, Fiona E; Harvey, John W; Brauer, Pamela P; Pirtle, Catherine P; Berríos, Kiara N; Newby, Gregory A; Yeh, Wei-Hsi; Kamath, Nikita; Mortberg, Meredith; Lian, Yuan; Howard, Michael; DeSouza-Lenz, Kendrick; Guzman, Kenia; Thai, Aaron; Graffam, Samantha; Laversenne, Vanessa; Coffey, Alissa A; Frei, Jeannine; Pierce, Sarah E; Safar, Jiri G; Deverman, Benjamin E; Minikel, Eric Vallabh; Vallabh, Sonia M; Liu, David R

In vivo base editing extends lifespan of a humanized mouse model of prion disease.

体内碱基编辑延长了朊病毒疾病人源化小鼠模型的寿命

An Meirui, Davis Jessie R, Levy Jonathan M, Serack Fiona E, Harvey John W, Brauer Pamela P, Pirtle Catherine P, Berríos Kiara N, Newby Gregory A, Yeh Wei-Hsi, Kamath Nikita, Mortberg Meredith, Lian Yuan, Howard Michael, DeSouza-Lenz Kendrick, Guzman Kenia, Thai Aaron, Graffam Samantha, Laversenne Vanessa, Coffey Alissa A, Frei Jeannine, Pierce Sarah E, Safar Jiri G, Deverman Benjamin E, Minikel Eric Vallabh, Vallabh Sonia M, Liu David R

Precisely defining disease variant effects in CRISPR-edited single cells

精确定义 CRISPR 编辑的单细胞中的疾病变异效应

Baglaenko, Yuriy; Mu, Zepeng; Curtis, Michelle; Mire, Hafsa M; Jayanthi, Vidyashree; Al Suqri, Majd; Liu, Cassidy; Agnew, Ryan; Nathan, Aparna; Mah-Som, Annelise Yoo; Liu, David R; Newby, Gregory A; Raychaudhuri, Soumya

Prime editing-installed suppressor tRNAs for disease-agnostic genome editing.

启动编辑,安装抑制性 tRNA,用于与疾病无关的基因组编辑。

Pierce Sarah E, Erwood Steven, Oye Keyede, An Meirui, Krasnow Nicholas, Zhang Emily, Raguram Aditya, Seelig Davis, Osborn Mark J, Liu David R

Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase

利用实验室进化的 CRISPR 相关转座酶在人类细胞中实现可编程基因插入

Witte, Isaac P; Lampe, George D; Eitzinger, Simon; Miller, Shannon M; Berríos, Kiara N; McElroy, Amber N; King, Rebeca T; Stringham, Olivia G; Gelsinger, Diego R; Vo, Phuc Leo H; Chen, Albert T; Tolar, Jakub; Osborn, Mark J; Sternberg, Samuel H; Liu, David R