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

Somatic deficiency of the human E3 ubiquitin ligase CBL in leukocytes impairs B cell but not T cell development and function.

白细胞中人类 E3 泛素连接酶 CBL 的体细胞缺陷会损害 B 细胞的发育和功能,但不会影响 T 细胞的发育和功能。

Vatovec Taja, Neehus Anna-Lena, Jackson Katherine J L, Avery Danielle T, Bagarić Ivan, Erazo Lucia, Arango-Franco Carlos A, Ogishi Masato, Ahmed Syed F, Cederholm Axel, Russell Amanda J, Della Mina Erika, Al-Rifai Dena, Bull Rowena, Buetow Lori, Sobrino Steicy, Zhang Allison, Wahlster Lara, Michelet Marine, Parvaneh Nima, Peel Jessica, Barzaghi Federica, Leardini Davide, Philippot Quentin, Saettini Francesco, Dutrieux Jacques, de Muylder Benedicte, Vendemini Francesca, Baccelli Francesco, Catala Albert, Gambineri Eleonora, Veltroni Marinella, Pandiarajan Vignesh, Aguilar Yurena, Haerynck Filomeen, Elliott Michael, Turville Stuart, Brillot Fabienne, Khan Taushif, Consonni Filippo, Berteloot Laureline, Sewell William A, Rao Geetha, Largeaud Laetitia, Conti Francesca, Roullion Cecile, Masson Cécile, Pegoraro Francesco, Ye Tianyi, Joubran Samantha, Villalpando Emily, Bessot Boris, Seeleuthner Yoann, Le Voyer Tom, Rosain Jérémie, Li Hailun, Janda Zarah, Muratore Edoardo, Soudée Camille, Delabesse Eric, Goulvestre Claire, Shahrooei Mohammad, Puel Anne, André Isabelle, Bole-Feysot Christine, Abel Laurent, Erlacher Miriam, Béziat Vivien, Lagresle-Peyrou Chantal, Cheynier Remi, Six Emmanuelle, Marr Nico, Pasquet Marlène, Alsina Laia, Goodnow Christopher C, Landegren Nils, Aiuti Alessandro, Zhang Peng, Masetti Riccardo, Huang Danny T, Ma Cindy S, Casanova Jean-Laurent, Sankaran Vijay G, Bustamante Jacinta, Tangye Stuart G, Bohlen Jonathan

Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells.

纳米工程化的 3D 培养基质能够使基因工程造血干细胞具有更强的持久性和多克隆移植能力。

Midena Federico, Alessandrini Laura, Conci Claudio, Barcella Matteo, Gazzo Francesco, Jacchetti Emanuela, Carsana Edoardo, Benedicenti Fabrizio, Vacca Roberta, Della Volpe Lucrezia, Arévalo Sergio, Giannetti Kety, Barozzi Dafne, Franchino Martina, Zonari Erika, Ferrua Francesca, Farina Giacomo, Brombin Chiara, Cugnata Federica, Fiumara Martina, Tavella Teresa, Cherubin Leonardo, Fraschetta Federico, Cerullo Giulio, Osellame Roberto, Radrizzani Marina, Ferrari Samuele, Merelli Ivan, Gentner Bernhard, Scielzo Cristina, Brendolan Andrea, Naldini Luigi, Aiuti Alessandro, Montini Eugenio, Raimondi Manuela T, Di Micco Raffaella

Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome

造血干细胞和祖细胞基因治疗(OTL-203)治疗赫勒综合征的非神经系统、非骨骼结局

Tucci, Francesca; Uria Oficialdegui, M Luz; Consiglieri, Giulia; Cossutta, Matilde; Filisetti, Chiara; Fumagalli, Francesca; Butera, Carla; Santangelo, Roberto; Colombo, Michele; Manitto, Maria Pia; Stoppani, Monica; Martina, Elisabetta; Danè, Giulia; Camesasca, Chiara; Risca, Giulia; De Pellegrin, Maurizio; Scarparo, Stefano; Sarzana, Marina; Puricelli, Cristina; Galimberti, Stefania; Darin, Silvia; Silvani, Paolo; Bonanomi, Sonia; Gasperini, Serena; Naldini, Luigi; Gentner, Bernhard; Parini, Rossella; Del Toro, Mireia; Diaz-de-Heredia, Cristina; Aiuti, Alessandro; Bernardo, Maria Ester

Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease

建立和表征IVA型粘多糖贮积症模型,用于评估针对骨骼疾病的疗法

Berti, Margherita; Ceriotti, Selene; Santi, Ludovica; Alberti, Gaia; Beretta, Stefano; Degl'Innocenti, Sara; Ruatti, Cristina; Savoia, Evelyn Oliva; Jofra-Hernandez, Raisa; De Ponti, Giada; Bolamperti, Simona; Villa, Isabella; Galeotti, Fabio; Romano, Alessandro; Visigalli, Ilaria; Norata, Rossana; Rocchi, Martina; Cristofori, Patrizia; Cossutta, Matilde; Consiglieri, Giulia; Tucci, Francesca; Santorelli, Lucia; Grumati, Paolo; Ronfani, Lorenza; D'Adamo, Patrizia; Giustina, Andrea; Angelozzi, Marco; Settembre, Carmine; Mortellaro, Alessandra; Scala, Serena; Sanvito, Francesca; Volpi, Nicola; Aiuti, Alessandro; Bernardo, Maria Ester; Crippa, Stefania

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

Mechanisms of hematopoietic clonal dominance in VEXAS syndrome.

VEXAS综合征中造血克隆优势的机制

Molteni Raffaella, Fiumara Martina, Campochiaro Corrado, Alfieri Roberta, Pacini Guido, Licari Eugenia, Tomelleri Alessandro, Diral Elisa, Varesi Angelica, Weber Alessandra, Quaranta Pamela, Albano Luisa, Gaddoni Chiara, Basso-Ricci Luca, Stefanoni Davide, Alessandrini Laura, Degl'Innocenti Sara, Sanvito Francesca, Bergonzi Gregorio Maria, Annoni Andrea, Panigada Maddalena, Cantoni Eleonora, Canarutto Daniele, Xie Stephanie Z, D'Alessandro Angelo, Di Micco Raffaella, Aiuti Alessandro, Ciceri Fabio, De Luca Giacomo, Dagna Lorenzo, Matucci-Cerinic Marco, Merelli Ivan, Cenci Simone, Scala Serena, Cavalli Giulio, Naldini Luigi, Ferrari Samuele

Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia

β-地中海贫血中TGFβ信号传导和自噬失衡驱动造血干细胞向红系分化。

Maria Rosa Lidonnici ,Giulia Chianella ,Nicole Mende ,Hugo P Bastos ,Matteo Barcella ,Ivan Merelli ,Mariangela Storto ,Valentina Romeo ,Francesca Tiboni ,Samantha Scaramuzza ,Claudia Rossi ,Laura Raggi ,Annamaria Aprile ,Stefania Crippa ,Deena Iskander ,Irene Roberts ,Anastasios Karamiditris ,Julia Keith ,Christophe Lechauve ,Mitchell J Weiss ,Nicola K Wilson ,Berthold Göttgens ,Maria Ester Bernardo ,Fabio Ciceri ,Alessandro Aiuti ,Sarah Marktel ,Elisa Laurenti ,Giuliana Ferrari

Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID

针对“基于造血干细胞的基因疗法治疗RAG1缺陷型重症联合免疫缺陷症的安全性和有效性考量”的回应

Montini, Eugenio; Naldini, Luigi; Booth, Claire; Kohn, Donald B; Aiuti, Alessandro

Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy

平衡慢病毒载体介导的造血干细胞基因治疗的疗效和安全性

Montini, Eugenio; Naldini, Luigi; Booth, Claire; Kohn, Donald B; Aiuti, Alessandro

Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities

腺苷脱氨酶2缺乏症患者早期骨髓改变与疾病表型和严重程度的关系

Dimitri Bulté ,Federica Barzaghi ,Cristina Mesa-Nuñez ,Chiara Rigamonti ,Luca Basso-Ricci ,Camilla Visconti ,Stefania Crippa ,Emanuela Pettinato ,Diego Gilioli ,Raffaella Milani ,Pamela Quaranta ,Roberta Caorsi ,Alessia Cafaro ,Giuliana Cangemi ,Michela Lupia ,Francesca Schena ,Alice Grossi ,Giulia Di Colo ,Silvia Federici ,Antonella Insalaco ,Fabrizio De Benedetti ,Sarah Marktel ,Raffaella Di Micco ,Maria Ester Bernardo ,Serena Scala ,Maria Pia Cicalese ,Francesca Conti ,Maurizio Miano ,Marco Gattorno ,Carlo Dufour ,Alessandro Aiuti ,Alessandra Mortellaro