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

Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.

临床前慢病毒造血干细胞基因疗法可纠正庞贝病相关的肌肉和神经系统症状

Yoon John K, Schindler Jeffrey W, Loperfido Mariana, Baricordi Cristina, DeAndrade Mark P, Jacobs Mary E, Treleaven Christopher, Plasschaert Robert N, Yan Aimin, Barese Cecilia N, Dogan Yildirim, Chen Vicky Ping, Fiorini Claudia, Hull Fritz, Barbarossa Luigi, Unnisa Zeenath, Ivanov Daniel, Kutner Robert H, Guda Swaroopa, Oborski Christine, Maiwald Tim, Michaud Véronique, Rothe Michael, Schambach Axel, Pfeifer Richard, Mason Chris, Biasco Luca, van Til Niek P

IS-Seq: a bioinformatics pipeline for integration sites analysis with comprehensive abundance quantification methods

IS-Seq:一种用于整合位点分析的生物信息学流程,包含全面的丰度定量方法

Yan, Aimin; Baricordi, Cristina; Nguyen, Quoc; Barbarossa, Luigi; Loperfido, Mariana; Biasco, Luca

Lentiviral gene therapy for X-linked chronic granulomatous disease

慢病毒基因疗法治疗X连锁慢性肉芽肿病

Kohn, Donald B; Booth, Claire; Kang, Elizabeth M; Pai, Sung-Yun; Shaw, Kit L; Santilli, Giorgia; Armant, Myriam; Buckland, Karen F; Choi, Uimook; De Ravin, Suk See; Dorsey, Morna J; Kuo, Caroline Y; Leon-Rico, Diego; Rivat, Christine; Izotova, Natalia; Gilmour, Kimberly; Snell, Katie; Dip, Jinhua Xu-Bayford; Darwish, Jinan; Morris, Emma C; Terrazas, Dayna; Wang, Leo D; Bauser, Christopher A; Paprotka, Tobias; Kuhns, Douglas B; Gregg, John; Raymond, Hayley E; Everett, John K; Honnet, Geraldine; Biasco, Luca; Newburger, Peter E; Bushman, Frederic D; Grez, Manuel; Gaspar, H Bobby; Williams, David A; Malech, Harry L; Galy, Anne; Thrasher, Adrian J

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

慢病毒造血干/祖细胞基因疗法治疗Wiskott-Aldrich综合征:一项非随机、开放标签的1/2期临床研究的中期结果

Ferrua, Francesca; Cicalese, Maria Pia; Galimberti, Stefania; Giannelli, Stefania; Dionisio, Francesca; Barzaghi, Federica; Migliavacca, Maddalena; Bernardo, Maria Ester; Calbi, Valeria; Assanelli, Andrea Angelo; Facchini, Marcella; Fossati, Claudia; Albertazzi, Elena; Scaramuzza, Samantha; Brigida, Immacolata; Scala, Serena; Basso-Ricci, Luca; Pajno, Roberta; Casiraghi, Miriam; Canarutto, Daniele; Salerio, Federica Andrea; Albert, Michael H; Bartoli, Antonella; Wolf, Hermann M; Fiori, Rossana; Silvani, Paolo; Gattillo, Salvatore; Villa, Anna; Biasco, Luca; Dott, Christopher; Culme-Seymour, Emily J; van Rossem, Koenraad; Atkinson, Gillian; Valsecchi, Maria Grazia; Roncarolo, Maria Grazia; Ciceri, Fabio; Naldini, Luigi; Aiuti, Alessandro

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients

在镰状细胞贫血患者中,单独使用普乐沙福成功动员和采集造血干细胞

Esrick, Erica B; Manis, John P; Daley, Heather; Baricordi, Cristina; Trébéden-Negre, Hélène; Pierciey, Francis J; Armant, Myriam; Nikiforow, Sarah; Heeney, Matthew M; London, Wendy B; Biasco, Luca; Asmal, Mohammed; Williams, David A; Biffi, Alessandra

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

在MNGIE小鼠模型中,造血干细胞基因疗法的临床前疗效和安全性评价

Yadak, Rana; Cabrera-Pérez, Raquel; Torres-Torronteras, Javier; Bugiani, Marianna; Haeck, Joost C; Huston, Marshall W; Bogaerts, Elly; Goffart, Steffi; Jacobs, Edwin H; Stok, Merel; Leonardelli, Lorena; Biasco, Luca; Verdijk, Robert M; Bernsen, Monique R; Ruijter, George; Martí, Ramon; Wagemaker, Gerard; van Til, Niek P; de Coo, Irenaeus F M

Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy

分析逆转录病毒载体在造血细胞基因治疗中的基因毒性

Biasco, Luca; Rothe, Michael; Büning, Hildegard; Schambach, Axel

Multiparametric Whole Blood Dissection: A one-shot comprehensive picture of the human hematopoietic system

多参数全血解剖:一次性全面了解人类造血系统

Basso-Ricci, Luca; Scala, Serena; Milani, Raffaella; Migliavacca, Maddalena; Rovelli, Attilio; Bernardo, Maria Ester; Ciceri, Fabio; Aiuti, Alessandro; Biasco, Luca

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome

慢病毒造血干细胞基因治疗在Wiskott-Aldrich综合征患者中的应用

Aiuti, Alessandro; Biasco, Luca; Scaramuzza, Samantha; Ferrua, Francesca; Cicalese, Maria Pia; Baricordi, Cristina; Dionisio, Francesca; Calabria, Andrea; Giannelli, Stefania; Castiello, Maria Carmina; Bosticardo, Marita; Evangelio, Costanza; Assanelli, Andrea; Casiraghi, Miriam; Di Nunzio, Sara; Callegaro, Luciano; Benati, Claudia; Rizzardi, Paolo; Pellin, Danilo; Di Serio, Clelia; Schmidt, Manfred; Von Kalle, Christof; Gardner, Jason; Mehta, Nalini; Neduva, Victor; Dow, David J; Galy, Anne; Miniero, Roberto; Finocchi, Andrea; Metin, Ayse; Banerjee, Pinaki P; Orange, Jordan S; Galimberti, Stefania; Valsecchi, Maria Grazia; Biffi, Alessandra; Montini, Eugenio; Villa, Anna; Ciceri, Fabio; Roncarolo, Maria Grazia; Naldini, Luigi

Retroviral integrations in gene therapy trials

基因治疗试验中的逆转录病毒整合

Biasco, Luca; Baricordi, Cristina; Aiuti, Alessandro