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

Monitoring biological effects of somatic cell genome editing

监测体细胞基因组编辑的生物学效应

Freedman, Benjamin S; Bulte, Jeff W M; Conklin, Bruce R; Judge, Luke M; Dwinell, Melinda R; Geurts, Aron M; Sitton, Madeleine J; Mahajan, Vineet; Kiani, Samira; Gersbach, Charles A; Ebrahimkhani, Mo R; Kelly, John J; Ronald, John A; Morizane, Ryuji; Gupta, Navin; Shakeri-Zadeh, Ali; Vo, Nicole; Saha, Krishanu; Saxena, Shivani; Gamm, David M; Sinha, Divya; Tarantal, Alice F; Vandsburger, Moriel; Matsubara, Azusa; Fu, Hongxia; Tsai, Shengdar Q

Measurement and clinical interpretation of CRISPR off-targets

CRISPR脱靶效应的测量和临床解读

Angelini Stewart, Ariella; Ahrens-Nicklas, Rebecca C; Tsai, Shengdar Q; Musunuru, Kiran; Giannikopoulos, Petros; Clelland, Claire D

GenomePAM directs PAM characterization and engineering of CRISPR-Cas nucleases using mammalian genome repeats

GenomePAM指导利用哺乳动物基因组重复序列对CRISPR-Cas核酸酶进行PAM表征和工程改造。

Yu, Miao; Ai, Limei; Wang, Bang; Lian, Shifeng; Ip, Lawrence; Liu, James; Li, Linxian; Tsai, Shengdar Q; Kleinstiver, Benjamin P; Zheng, Zongli

Engineering a compact high-fidelity Staphylococcus aureus Cas9 variant with broader targeting range and mechanistic insights into its activation

构建一种结构紧凑、高保真度的金黄色葡萄球菌Cas9变体,使其具有更广泛的靶向范围,并深入了解其激活机制。

Omura, Satoshi N; Nakagawa, Ryoya; Kajimoto, Shohei; Okazaki, Sae; Ishiguro, Soh; Mori, Hideto; Onishi, Kosuke; Kashiwakura, Yuji; Hiramoto, Takafumi; Horinaka, Kio; Tanaka, Mamoru; Hirano, Hisato; Jividen, Kasey; Yamashita, Keitaro; Tsai, Shengdar Q; Yachie, Nozomu; Ohmori, Tsukasa; Nishimasu, Hiroshi; Nureki, Osamu

Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning

通过可扩展的工程和机器学习技术构建定制的 CRISPR-Cas9 PAM 变体

Silverstein, Rachel A; Kim, Nahye; Kroell, Ann-Sophie; Walton, Russell T; Delano, Justin; Butcher, Rossano M; Pacesa, Martin; Smith, Blaire K; Christie, Kathleen A; Ha, Leillani L; Meis, Ronald J; Clark, Aaron B; Spinner, Aviv D; Lazzarotto, Cicera R; Li, Yichao; Matsubara, Azusa; Urbina, Elizabeth O; Dahl, Gary A; Correia, Bruno E; Marks, Debora S; Tsai, Shengdar Q; Pinello, Luca; De Ravin, Suk See; Liu, Qin; Kleinstiver, Benjamin P

Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.

利用定制的 CRISPR-Cas9 碱基编辑器治疗小鼠的严重血管疾病。

Alves Christiano R R, Das Sabyasachi, Krishnan Vijai, Ha Leillani L, Fox Lauren R, Stutzman Hannah E, Shamber Claire E, Kalailingam Pazhanichamy, McCarthy Siobhan, Lino Cardenas Christian L, Fong Claire E, Imai Takahiko, Mitra Sunayana, Yun Shuqi, Wood Rachael K, Benning Friederike M C, Roh Kangsan, Lawton Joseph, Kim Nahye, Silverstein Rachel A, Ferreira da Silva Joana, de la Cruz Demitri, Richa Rashmi, Xie Jun, Gray-Edwards Heather L, Malhotra Rajeev, Chung David Y, Chao Luke H, Tsai Shengdar Q, Maguire Casey A, Lindsay Mark E, Kleinstiver Benjamin P, Musolino Patricia L

Virus-free CRISPR knockin of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells.

利用无病毒 CRISPR 技术将嵌合抗原受体敲入 KLRC1 中,可产生强效的 GD2 特异性自然杀伤细胞

Shankar Keerthana, Zingler-Hoslet Isabelle, Tabima Diana M, Zima Seth, Shi Lei, Gimse Kirstan, Forsberg Matthew H, Katta Varun, Davis Sage Z, Maldonado Daniel, Russell Brittany E, Murtaza Muhammed, Tsai Shengdar Q, Ayuso Jose M, Capitini Christian M, Saha Krishanu

Population-scale cellular GUIDE-seq-2 and biochemical CHANGE-seq-R profiles reveal human genetic variation frequently affects Cas9 off-target activity

群体规模的细胞 GUIDE-seq-2 和生化 CHANGE-seq-R 分析表明,人类遗传变异经常影响 Cas9 的脱靶活性。

Lazzarotto, Cicera R; Li, Yichao; Flory, Ashley R; Chyr, Jacqueline; Yang, Muyu; Katta, Varun; Urbina, Elizabeth; Lee, GaHyun; Wood, Rachael; Matsubara, Azusa; Rashkin, Sara R; Ma, Jian; Cheng, Yong; Tsai, Shengdar Q

Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome

利用STEP-RNP进行全脑基因组编辑治疗安格曼综合征

Lu, Xiaona; Xie, Ying; Bao, Youmei; Yu, Jiang; Wang, Zefeng; Fan, Jiali; Sheu, Wendy C; Long, Gretchen; Tu, Zewei; Tong, Jie; Manquen, Garret; Li, Yichao; Katta, Varun; Chow, Ryan; Chen, Binfan; Meng, Zhouqi; Ma, Ying-Hong; Qiu, Caihong; Chen, Sidi; Tsai, Shengdar Q; De Kumar, Bony; Hendry, Caroline E; Jiang, Yong-Hui; Zhou, Jiangbing

Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

优化碱基编辑器,用于功能性校正 SMN2 以治疗脊髓性肌萎缩症

Christiano R R Alves, Leillani L Ha, Rebecca Yaworski, Emma R Sutton, Cicera R Lazzarotto, Kathleen A Christie, Aoife Reilly, Ariane Beauvais, Roman M Doll, Demitri de la Cruz, Casey A Maguire, Kathryn J Swoboda, Shengdar Q Tsai, Rashmi Kothary, Benjamin P Kleinstiver