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

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

CRISPR/Cas9 Screens Implicate RARA and SPNS1 in Doxorubicin Cardiotoxicity

CRISPR/Cas9筛选表明RARA和SPNS1与阿霉素心脏毒性有关

McDermott-Roe, Chris; Lv, Wenjian; Shao, Yeng; Hoshino, Atsushi; Arany, Zoltan; Musunuru, Kiran

Response to the FDA's proposed pathway for individualized genetic therapies

对FDA提出的个体化基因治疗途径的回应

Yu, Timothy W; Vitarello, Julia; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C; Liu, David R; Mellion, Michelle L; Urnov, Fyodor; Yan, Winston; Chunduru, Srinivas; Barrett, David; Flotte, Terence R; Woodcock, Janet

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

Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing

利用腺嘌呤碱基编辑技术纠正甲基丙二酸血症中的复发性致病变异

Kahn, Elena M; Said, Hooda; Qu, Ping; Alameh, Mohamad-Gabriel; Wang, Xiao; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C

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

Breakthroughs in targeted in vivo gene editing: Overcoming barriers to effective delivery

靶向体内基因编辑的突破:克服有效递送的障碍

Cannon, Paula; Musunuru, Kiran

Systemic delivery of biotherapeutic RNA to the myocardium transiently modulates cardiac contractility in vivo.

将生物治疗性 RNA 系统性输送至心肌可暂时性地调节体内心脏收缩力

Shuvaev Vladimir V, Tam Ying K, Lee Benjamin W, Myerson Jacob W, Herbst Alan, Kiseleva Raisa Yu, Glassman Patrick M, Parhiz Hamideh, Alameh Mohamad-Gabriel, Pardi Norbert, Muramatsu Hiromi, Shuvaeva Tea I, Arguiri Evguenia, Marcos-Contreras Oscar A, Hood Elizabeth D, Brysgel Taylor V, Nong Jia, Papp Tyler E, Eaton Deborah M, Riley Rachel, Palanki Rohan, Musunuru Kiran, Brenner Jacob S, Mitchell Michael J, Ferrari Victor A, Mui Barbara L, Semple Sean C, Weppler Sherry A, Atluri Pavan, Margulies Kenneth B, Weissman Drew, Muzykantov Vladimir R

Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants

针对两种高复发性苯丙酮尿症变异体的体内碱基编辑纠正策略

Quigley, Aidan; Jindal, Ishaan; Campion, Thomas; Rutherford, Delaney; Han, Yongseok; Quigley, Walsh; Qu, Ping; Musunuru, Kiran; Ahrens-Nicklas, Rebecca; Hong, Xinying; Wang, Xiao

Liver-directed base editing of ABCC6 prevents ectopic calcification in a variant-humanized mouse model of pseudoxanthoma elasticum.

针对肝脏的 ABCC6 碱基编辑可防止假性黄色瘤人源化小鼠模型中的异位钙化。

Testa Lauren C, Obiri-Yeboah Dora, Said Hooda, Qu Ping, Levine Michael A, Alameh Mohamad-Gabriel, Musunuru Kiran, Li Qiaoli, Wang Xiao