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

In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking

出生后干细胞迁移促进体内造血干细胞基因治疗

Michela Milani ,Anna Fabiano ,Marta Perez-Rodriguez ,Raisa Jofra Hernandez ,Alessandra Zecchillo ,Erika Zonari ,Sofia Ottonello ,Luca Basso-Ricci ,Cesare Canepari ,Monica Volpin ,Valeria Iannello ,Valentina Capo ,Pamela Quaranta ,Luca Seffin ,Fabio Russo ,Mauro Biffi ,Leonardo Ormoli ,Chiara Brombin ,Filippo Carlucci ,Antonella Forlino ,Marta Filibian ,Eugenio Montini ,Serena Scala ,Anna Villa ,Juan Antonio Bueren ,Paula Rio ,Alessandro Aiuti ,Alessio Cantore ,Luigi Naldini

Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes

增强体内慢病毒载体介导的基因治疗对肝细胞的效力

Cesare Canepari,Michela Milani,Chiara Simoni,Francesco Starinieri,Monica Volpin,Anna Fabiano,Mauro Biffi,Fabio Russo,Rossana Norata,Martina Rocchi,Chiara Brombin,Federica Cugnata,Eugenio Montini,Francesca Sanvito,Markus Grompe,Alessio Cantore

Identification of hepatocyte-primed cholangiocytes in the homeostatic liver by in vivo lentiviral gene transfer to mice and non-human primates

通过体内慢病毒基因转移至小鼠和非人灵长类动物,鉴定稳态肝脏中肝细胞启动的胆管细胞。

Michela Milani,Francesco Starinieri,Anna Fabiano,Stefano Beretta,Tiziana Plati,Cesare Canepari,Mauro Biffi,Fabio Russo,Valeria Berno,Rossana Norata,Francesca Sanvito,Ivan Merelli,Luigi Aloia,Meritxell Huch,Luigi Naldini,Alessio Cantore

Identification of hepatocyte-primed cholangiocytes in the homeostatic liver by in vivo lentiviral gene transfer to mice and non-human primates

通过体内慢病毒基因转移至小鼠和非人灵长类动物,鉴定稳态肝脏中肝细胞启动的胆管细胞。

Michela Milani ,Francesco Starinieri ,Anna Fabiano ,Stefano Beretta ,Tiziana Plati ,Cesare Canepari ,Mauro Biffi ,Fabio Russo ,Valeria Berno ,Rossana Norata ,Francesca Sanvito ,Ivan Merelli ,Luigi Aloia ,Meritxell Huch ,Luigi Naldini ,Alessio Cantore

GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice

GP64 假型慢病毒载体靶向肝内皮细胞并纠正血友病 A 小鼠

Michela Milani, Cesare Canepari, Simone Assanelli, Simone Merlin, Ester Borroni, Francesco Starinieri, Mauro Biffi, Fabio Russo, Anna Fabiano, Desirèe Zambroni, Andrea Annoni, Luigi Naldini, Antonia Follenzi, Alessio Cantore

Thyroid Function and Metabolic Syndrome in Children and Adolescents with Neuromotor Disability

甲状腺功能与神经运动障碍儿童和青少年代谢综合征

Calcaterra, Valeria; Biganzoli, Giacomo; Ferraro, Simona; Mari, Alessandra; Mandelli, Anna; Fabiano, Valentina; Carlucci, Patrizia; Pelizzo, Gloria; Zoia, Elena; Lanfranchi, Giulia; Castaldi, Silvana; Boracchi, Patrizia; Biganzoli, Elia; Zuccotti, Gianvincenzo

Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

通过 CRISPR/Cas9 编辑人类造血干细胞中的 α-珠蛋白基因座来纠正 β-地中海贫血

Giulia Pavani, Anna Fabiano, Marine Laurent, Fatima Amor, Erika Cantelli, Anne Chalumeau, Giulia Maule, Alexandra Tachtsidi, Jean-Paul Concordet, Anna Cereseto, Fulvio Mavilio, Giuliana Ferrari, Annarita Miccio, Mario Amendola

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

IgG 裂解内肽酶可在抗 AAV 中和抗体存在的情况下进行体内基因治疗

Christian Leborgne #, Elena Barbon #, Jeffrey M Alexander #, Hayley Hanby, Sandrine Delignat, Daniel M Cohen, Fanny Collaud, Saghana Muraleetharan, Dan Lupo, Joseph Silverberg, Karen Huang, Laetitia van Wittengerghe, Béatrice Marolleau, Adeline Miranda, Anna Fabiano, Victoria Daventure, Heena Beck, 

Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

体外编辑人造血干细胞以使其在红系中表达治疗性蛋白

Giulia Pavani,Marine Laurent,Anna Fabiano,Erika Cantelli,Aboud Sakkal,Guillaume Corre,Peter J Lenting,Jean-Paul Concordet,Magali Toueille,Annarita Miccio,Mario Amendola