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

Nucleotide-resolution mapping of regulatory elements via allelic readout of tiled base editing.

通过等位基因读取平铺碱基编辑,实现调控元件的核苷酸分辨率定位。

Becerra Basheer, Wittibschlager Sandra, Patel Zain M, Kutschat Ana P, Delano Justin, Che Eric, Tauber Anzhelika, Wu Ting, Starrs Marlena, Horstmann Christina S, Müller Sophie, Whittaker Madelynn N, Sylvander Elise, Lehner Manfred, Love Michael I, Kleinstiver Benjamin P, Jankowiak Martin, Bauer Daniel E, Seruggia Davide, Pinello Luca

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

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

Engineering of CD63 Enables Selective Extracellular Vesicle Cargo Loading and Enhanced Payload Delivery.

CD63 的工程改造可实现选择性细胞外囊泡货物装载和增强有效载荷递送

Obuchi Wataru, Zargani-Piccardi Ayrton, Leandro Kevin, Rufino-Ramos David, Di Lanni Emilio, Frederick Dawn Madison, Maalouf Katia, Nieland Lisa, Xiao Tianhe, Repiton Pierre, Vaine Christine A, Kleinstiver Benjamin P, Bragg D Cristopher, Lee Hakho, Miller Miles A, Breakefield Xandra O, Breyne Koen

CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice

利用单个腺相关病毒载体进行 CRISPR 靶向 mmu-miR-21a 可延长胶质母细胞瘤小鼠的生存期。

Lisa Nieland ,Anne B Vrijmoet ,Isabelle W Jetten ,David Rufino-Ramos ,Alexandra J E M de Reus ,Koen Breyne ,Benjamin P Kleinstiver ,Casey A Maguire ,Marike L D Broekman ,Xandra O Breakefield ,Erik R Abels

Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of TOR1A DYT1 allele in a xenograft mouse model

在异种移植小鼠模型中,通过非侵入性方法检测 TOR1A DYT1 等位基因的等位基因特异性 CRISPR-SaCas9-KKH 破坏

Katia E Maalouf ,Dawn Madison Frederick ,Nutan Sharma ,Edwina Abou Haidar ,Tianhe Xiao ,Justin Seungkyu Han ,Mohammed S Mahamdeh ,Roy J Soberman ,David Rufino-Ramos ,Benjamin P Kleinstiver ,Hyder A Jinnah ,Christine A Vaine ,D Cristopher Bragg ,Koen Breyne

An opportune time for targeted brain arteriovenous malformation therapy

针对脑动静脉畸形进行靶向治疗的良机

Hale, Andrew T; Kalailingam, Pazhanichamy; Kundishora, Adam J; Reeves, Benjamin C; Brastianos, Priscilla K; Shih, Helen A; Jain, Rakesh K; Ning, MingMing; Stapleton, Christopher J; Patel, Aman B; Smith, Edward R; Chapman, Paul H; Rabinov, James D; Kleinstiver, Benjamin P; Musolino, Patricia L; Kahle, Kristopher T

Engineering of CRISPR-Cas PAM recognition using deep learning of vast evolutionary data

利用深度学习对海量进化数据进行CRISPR-Cas PAM识别的工程改造

Nayfach, Stephen; Bhatnagar, Aadyot; Novichkov, Andrey; Estevam, Gabriella O; Kim, Nahye; Hill, Emily; Ruffolo, Jeffrey A; Silverstein, Rachel; Gallagher, Joseph; Kleinstiver, Benjamin; Meeske, Alexander J; Cameron, Peter; Madani, Ali