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

Autoantibodies neutralizing type I IFNs in 40% of patients with WNV encephalitis in seven new cohorts

在七个新的队列研究中,40%的西尼罗河病毒脑炎患者体内存在中和I型干扰素的自身抗体。

Gervais, Adrian; Trespidi, Francesca; Ferrari, Alessandro; Rovida, Francesca; Marchal, Astrid; Croce, Stefania; Cassaniti, Irene; Moratti, Mattia; Uhrlaub, Jennifer L; Florian, David M; Stiasny, Karin; Burdino, Elisa; Angelini, Micol; Bizien, Lucy; Lilleri, Daniele; Codullo, Veronica; Freund, Tal; Paran, Yael; Gadoth, Avi; Biran, Roni; Mancon, Alessandro; Lucca, Camilla; Vogiatzis, Stefania; Pacenti, Monia; Aubart, Mélodie; Zecca, Marco; Comoli, Patrizia; Avanzini, Maria Antonietta; Fellay, Jacques; Piralla, Antonio; Conti, Francesca; Dolci, Alberto; Barzon, Luisa; Ghisetti, Valeria; Lazzarotto, Tiziana; Cereda, Danilo; Aiuti, Alessandro; Jouanguy, Emmanuelle; Bastard, Paul; MacDonald, Margaret R; Rice, Charles M; Puel, Anne; Abel, Laurent; Rossini, Giada; Mileto, Davide; Simonin, Yannick; Nagy, Anna; Hagin, David; Murray, Kristy O; Baldanti, Fausto; Aberle, Judith H; Cobat, Aurélie; Zhang, Shen-Ying; Casanova, Jean-Laurent; Borghesi, Alessandro

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

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

Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET)

Wiskott-Aldrich 综合征和 X 连锁血小板减少症患者的长期预后:意大利原发性免疫缺陷网络 (IPINET) 的一项观察性前瞻性多中心研究

Soresina, Annarosa; Rondelli, Roberto; Notarangelo, Lucia Dora; Locatelli, Franco; Aiuti, Alessandro; Biffi, Alessandra; Rabusin, Marco; Pignata, Claudio; Menna, Giuseppe; Prete, Arcangelo; Faraci, Maura; Rovelli, Attilio; Conti, Francesca; Bertolini, Patrizia; Azzari, Chiara; Cancrini, Caterina; Zecca, Marco; Ferrua, Francesca; Cicalese, Maria Pia; Cecere, Francesco; Dotta, Laura; Martire, Baldassarre; Giliani, Silvia; Moratto, Daniele; Mazza, Cinzia; Plebani, Alessandro; Notarangelo, Luigi D; Pession, Andrea; Badolato, Raffaele; Fulvio, Fulvio

Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I

新生儿基因疗法可有效预防小鼠I型粘多糖贮积症模型中的疾病表现。

De Ponti, Giada; Santi, Ludovica; Dina, Giorgia; Pievani, Alice; Donsante, Samantha; Riminucci, Mara; Corsi, Alessandro; Khan, Shaukat; Passerini, Laura; Annoni, Andrea; Gregori, Silvia; Crippa, Stefania; Biondi, Andrea; Quattrini, Angelo; Tomatsu, Shunji; Aiuti, Alessandro; Bernardo, Maria Ester; Serafini, Marta