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C

Cícera R. Lazzarotto

st jude childrens research hospital
H指数:20
被引数:3,277

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A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy

一种精确基因工程B细胞药物，可持续产生活性因子IX，用于血友病B的治疗

Molecular Therapy
IF12
2025-09-050
OAAI
Hanlan Liu; Swati Singh; Timothy J. Mullen; Caroline Bullock; Sean Keegan; Troy Patterson; Sakshisingh Thakur; Amy Lundberg; Sol Shenker; Ryan Couto; Charuta Yadav; Shamael Dastagir; Lily Li; Wayne Bainter; Ella Liberzon; Connor R. Malloy; Cicera R. Lazzarotto; Toshiro K. Ohsumi; Shalini Chilakala; Huei-Mei Chen; Rashmi Kshirsagar; Anja F. Hohmann; Sean Arlauckas; Adam Lazorchak; Chris Scull; Richard A. Morgan
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease

造血干细胞的高保真无PAMless碱基编辑治疗慢性肉芽肿病

SCIENCE TRANSLATIONAL MEDICINE
IF14.6
2024-10-160
PRE
Bzhilyanskaya, Vera; Ma, Linyuan; Liu, Siyuan; Fox, Lauren R.; Whittaker, Madelynn N.; Meis, Ronald J.; Choi, Uimook; Lawson, Amanda; Ma, Michelle; Theobald, Narda; Burkett, Sandra; Sweeney, Colin L.; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Lack, Justin B.; Wu, Xiaolin; Dahl, Gary A.; Malech, Harry L.; Kleinstiver, Benjamin P.; De Ravin, Suk See
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+cell therapy to induce fetal hemoglobin for sickle cell disease

新型Cas9基因组编辑的自体CD34细胞疗法诱导胎儿血红蛋白治疗镰状细胞病的开发和IND支持研究

MOLECULAR THERAPY
IF12
2024-10-010
OAAI
Katta, Varun; O'Keefe, Kiera; Li, Yichao; Mayuranathan, Thiyagaraj; Lazzarotto, Cicera R.; Wood, Rachael K.; Levine, Rachel M.; Powers, Alicia; Mayberry, Kalin; Manquen, Garret; Yao, Yu; Zhang, Jingjing; Jang, Yoonjeong; Nimmagadda, Nikitha; Dempsey, Erin A.; Lee, GaHyun; Uchida, Naoya; Cheng, Yong; Fazio, Frank; Lockey, Tim; Meagher, Mike; Sharma, Akshay; Tisdale, John F.; Zhou, Sheng; Yen, Jonathan S.; Weiss, Mitchell J.; Tsai, Shengdar Q.
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

用于SMN2功能矫正的碱基编辑器的优化，以治疗脊髓性肌萎缩症

NATURE BIOMEDICAL ENGINEERING
IF26.6
2023-12-065
OA
Alves, Christiano R. R.; Ha, Leillani L.; Yaworski, Rebecca; Sutton, Emma R.; Lazzarotto, Cicera R.; Christie, Kathleen A.; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M.; de la Cruz, Demitri; Maguire, Casey A.; Swoboda, Kathryn J.; Tsai, Shengdar Q.; Kothary, Rashmi; Kleinstiver, Benjamin P.
Potent and uniform fetal hemoglobin induction via base editing
NATURE GENETICS
IF31.8
2023-07-0325
OAAI
Mayuranathan, Thiyagaraj; Newby, Gregory A.; Feng, Ruopeng; Yao, Yu; Mayberry, Kalin D.; Lazzarotto, Cicera R.; Li, Yichao; Levine, Rachel M.; Nimmagadda, Nikitha; Dempsey, Erin; Kang, Guolian; Porter, Shaina N.; Doerfler, Phillip A.; Zhang, Jingjing; Jang, Yoonjeong; Chen, Jingjing; Bell, Henry W.; Crossley, Merlin; Bhoopalan, Senthil Velan; Sharma, Akshay; Tisdale, John F.; Pruett-Miller, Shondra M.; Cheng, Yong; Tsai, Shengdar Q.; Liu, David R.; Weiss, Mitchell J.; Yen, Jonathan S.
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells
FRONTIERS IN IMMUNOLOGY
IF5.9
2023-01-0418
OAAI
Brault, Julie; Liu, Taylor; Liu, Siyuan; Lawson, Amanda; Choi, Uimook; Kozhushko, Nikita; Bzhilyanskaya, Vera; Pavel-Dinu, Mara; Meis, Ronald J.; Eckhaus, Michael A.; Burkett, Sandra S.; Bosticardo, Marita; Kleinstiver, Benjamin P.; Notarangelo, Luigi D.; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Wu, Xiaolin; Dahl, Gary A.; Porteus, Matthew H.; Malech, Harry L.; De Ravin, Suk See
Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression

能够诱导实体瘤消退的无病毒crispr-car T细胞的产生和表征

JOURNAL FOR IMMUNOTHERAPY OF CANCER
IF10.6
2022-09-0728
OAAI
Mueller, Katherine P.; Piscopo, Nicole J.; Forsberg, Matthew H.; Saraspe, Louise A.; Das, Amritava; Russell, Brittany; Smerchansky, Madeline; Cappabianca, Dan; Shi, Lei; Shankar, Keerthana; Sarko, Lauren; Khajanchi, Namita; Denne, Nina La Vonne; Ramamurthy, Apoorva; Ali, Adeela; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Capitini, Christian M.; Saha, Krishanu
Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques
MOLECULAR THERAPY
IF12
2022-01-012
OA
AlJanahi, Aisha A.; Lazzarotto, Cicera R.; Chen, Shirley; Shin, Tae-Hoon; Cordes, Stefan; Fan, Xing; Jabara, Isabel; Zhou, Yifan; Young, David J.; Lee, Byung-Chul; Yu, Kyung-Rok; Li, Yuesheng; Toms, Bradley; Tunc, Ilker; Hong, So Gun; Truitt, Lauren L.; Klermund, Julia; Andrieux, Geoffroy; Kim, Miriam Y.; Cathomen, Toni; Gill, Saar; Tsai, Shengdar Q.; Dunbar, Cynthia E.
Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity

在CAR T细胞中删除DNMT3A可防止耗竭并增强抗肿瘤活性

SCIENCE TRANSLATIONAL MEDICINE
IF14.6
2021-11-17180
OA
Prinzing, Brooke; Zebley, Caitlin C.; Petersen, Christopher T.; Fan, Yiping; Anido, Alejandro Allo; Yi, Zhongzhen; Phuong Nguyen; Houke, Haley; Bell, Matthew; Haydar, Dalia; Brown, Charmaine; Boi, Shannon K.; Alli, Shanta; Crawford, Jeremy Chase; Riberdy, Janice M.; Park, Jeoungeun J.; Zhou, Sheng; Velasquez, Mireya Paulina; DeRenzo, Chris; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Vogel, Peter; Pruett-Miller, Shondra M.; Langfitt, Deanna M.; Gottschalk, Stephen; Youngblood, Ben; Krenciute, Giedre
Base editing of haematopoietic stem cells rescues sickle cell disease in mice
Nature
IF48.5
2021-06-020
PREAI
Gregory A. Newby; Jonathan S. Yen; Kaitly J. Woodard; Thiyagaraj Mayuranathan; Cicera R. Lazzarotto; Yichao Li; Heather Sheppard-Tillman; Shaina N. Porter; Yu Yao; Kalin Mayberry; Kelcee A. Everette; Yoonjeong Jang; Christopher J. Podracky; Elizabeth Thaman; Christophe Lechauve; Akshay Sharma; Jordana M. Henderson; Michelle F. Richter; Kevin T. Zhao; Shannon M. Miller; Tina Wang; Luke W. Koblan; Anton P. McCaffrey; John F. Tisdale; Theodosia A. Kalfa; Shondra M. Pruett-Miller; Shengdar Q. Tsai; Mitchell J. Weiss; David R. Liu
Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression

小鼠中的Prime编辑揭示了单个碱基在驱动组织特异性基因表达中的重要性

GENOME BIOLOGY
IF9.4
2021-03-1679
OAAI
Gao, Pan; Lyu, Qing; Ghanam, Amr R.; Lazzarotto, Cicera R.; Newby, Gregory A.; Zhang, Wei; Choi, Mihyun; Slivano, Orazio J.; Holden, Kevin; Walker, John A., II; Kadina, Anastasia P.; Munroe, Rob J.; Abratte, Christian M.; Schimenti, John C.; Liu, David R.; Tsai, Shengdar Q.; Long, Xiaochun; Miano, Joseph M.
BCL11A enhancer edited hematopoietic stem cells persist in rhesus monkeys without toxicity

BCL11A增强子编辑的造血干细胞在恒河猴中持续存在而没有毒性

JOURNAL OF CLINICAL INVESTIGATION
IF13.6
2020-11-0953
OA
Demirci, Selami; Zeng, Jing; Wu, Yuxuan; Uchida, Naoya; Shen, Anne H.; Pellin, Danilo; Gamer, Jackson; Yapundich, Morgan; Drysdale, Claire; Bonanno, Jasmine; Bonifacino, Aylin C.; Krouse, Allen E.; Linde, Nathaniel S.; Engels, Theresa; Donahue, Robert E.; Haro-Mora, Juan J.; Leonard, Alexis; Nassehi, Tina; Luk, Kevin; Porter, Shaina N.; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Weiss, Mitchell J.; Pruett-Miller, Shondra M.; Wolfe, Scot A.; Bauer, Daniel E.; Tisdale, John F.
CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity

CHANGE-seq揭示了遗传和表观遗传对CRISPR-Cas9全基因组活性的影响

NATURE BIOTECHNOLOGY
IF41.7
2020-06-15149
OAAI
Lazzarotto, Cicera R.; Malinin, Nikolay L.; Li, Yichao; Zhang, Ruochi; Yang, Yang; Lee, GaHyun; Cowley, Eleanor; He, Yanghua; Lan, Xin; Jividen, Kasey; Katta, Varun; Kolmakova, Natalia G.; Petersen, Christopher T.; Qi, Qian; Strelcov, Evgheni; Maragh, Samantha; Krenciute, Giedre; Ma, Jian; Cheng, Yong; Tsai, Shengdar Q.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
BLOOD ADVANCES
IF7.1
2019-11-06132
OA
Metais, Jean-Yves; Doerfler, Phillip A.; Mayuranathan, Thiyagaraj; Bauer, Daniel E.; Fowler, Stephanie C.; Hsieh, Matthew M.; Katta, Varun; Keriwala, Sagar; Lazzarotto, Cicera R.; Luk, Kevin; Neel, Michael D.; Perry, S. Scott; Peters, Samuel T.; Porter, Shaina N.; Ryu, Byoung Y.; Sharma, Akshay; Shea, Devlin; Tisdale, John F.; Uchida, Naoya; Wolfe, Scot A.; Woodard, Kaitly J.; Wu, Yuxuan; Yao, Yu; Zeng, Jing; Pruett-Miller, Shondra; Tsai, Shengdar Q.; Weiss, Mitchell J.
Highly efficient therapeutic gene editing of human hematopoietic stem cells

人类造血干细胞的高效治疗性基因编辑

NATURE MEDICINE
IF50
2019-03-25365
OAAI
Wu, Yuxuan; Zeng, Jing; Roscoe, Benjamin P.; Liu, Pengpeng; Yao, Qiuming; Lazzarotto, Cicera R.; Clement, Kendell; Cole, Mitchel A.; Luk, Kevin; Baricordi, Cristina; Shen, Anne H.; Ren, Chunyan; Esrick, Erica B.; Manis, John P.; Dorfman, David M.; Williams, David A.; Biffi, Alessandra; Brugnara, Carlo; Biasco, Luca; Brendel, Christian; Pinello, Luca; Tsai, Shengdar Q.; Wolfe, Scot A.; Bauer, Daniel E.

研究方向

暂未获取到该数据
S
Shengdar Q. Tsai
H 指数:49论文数:150被引数:2.2万
Y
Yichao Li
H 指数:27论文数:143被引数:2,687
M
Mitchell J. Weiss
H 指数:87论文数:451被引数:2.7万
S
Shondra M. Pruett‐Miller
H 指数:48论文数:377被引数:9,051
J
John F. Tisdale
H 指数:62论文数:436被引数:1.6万
G
GaHyun Lee
H 指数:9论文数:17被引数:592
A
Akshay Sharma
H 指数:31论文数:430被引数:6,823
V
Varun Katta
H 指数:8论文数:21被引数:665
J
Jing Zeng
H 指数:28论文数:143被引数:4,484
S
Suk See De Ravin
H 指数:44论文数:152被引数:8,115