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

Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency

造血干细胞和祖细胞中的外显子敲除和敲入基因编辑可挽救 RAG1 免疫缺陷

Maria Carmina Castiello, Chiara Brandas, Samuele Ferrari, Simona Porcellini, Nicolò Sacchetti, Daniele Canarutto, Elena Draghici, Ivan Merelli, Matteo Barcella, Gabriele Pelosi, Valentina Vavassori, Angelica Varesi, Aurelien Jacob, Serena Scala, Luca Basso Ricci, Marianna Paulis, Dario Strina, Marti

Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1

对基因组完整性进行无偏评估,并在编辑 CD4+ T 细胞治疗高 IgM1 综合征时清除靶位点的不良结果

Daniele Canarutto ,Claudia Asperti ,Valentina Vavassori ,Simona Porcellini ,Elisabetta Rovelli ,Marianna Paulis ,Samuele Ferrari ,Angelica Varesi ,Martina Fiumara ,Aurelien Jacob ,Lucia Sergi Sergi ,Ilaria Visigalli ,Francesca Ferrua ,Luis Ignacio González-Granado ,Vassilios Lougaris ,Andrea Finocchi ,Anna Villa ,Marina Radrizzani ,Luigi Naldini

Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo

使用 IDLV 模板对 CD4+ T 细胞进行可扩展且符合 GMP 标准的基因校正,并在体内和体外进行了功能验证

Claudia Asperti, Daniele Canarutto, Simona Porcellini, Francesca Sanvito, Francesca Cecere, Valentina Vavassori, Samuele Ferrari, Elisabetta Rovelli, Luisa Albano, Aurelien Jacob, Lucia Sergi Sergi, Elisa Montaldo, Francesca Ferrua, Luis Ignacio González-Granado, Vassilios Lougaris, Raffaele Badolat

Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells

基于动员的无化疗基因编辑人造血干细胞移植

Attya Omer-Javed ,Gabriele Pedrazzani ,Luisa Albano ,Sherash Ghaus ,Claire Latroche ,Maura Manzi ,Samuele Ferrari ,Martina Fiumara ,Aurelien Jacob ,Valentina Vavassori ,Alessandro Nonis ,Daniele Canarutto ,Luigi Naldini

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

模板递送的选择可减轻人类造血干细胞编辑的基因毒性风险和不利影响

Samuele Ferrari, Aurelien Jacob, Daniela Cesana, Marianne Laugel, Stefano Beretta, Angelica Varesi, Giulia Unali, Anastasia Conti, Daniele Canarutto, Luisa Albano, Andrea Calabria, Valentina Vavassori, Carlo Cipriani, Maria Carmina Castiello, Simona Esposito, Chiara Brombin, Federica Cugnata, Oumeya

BAR-Seq clonal tracking of gene-edited cells

BAR-Seq 基因编辑细胞的克隆追踪

Samuele Ferrari #, Stefano Beretta #, Aurelien Jacob, Davide Cittaro, Luisa Albano, Ivan Merelli, Luigi Naldini, Pietro Genovese

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

通过克隆追踪验证人类长期造血干细胞的有效基因编辑

Samuele Ferrari #, Aurelien Jacob #, Stefano Beretta, Giulia Unali, Luisa Albano, Valentina Vavassori, Davide Cittaro, Dejan Lazarevic, Chiara Brombin, Federica Cugnata, Anna Kajaste-Rudnitski, Ivan Merelli, Pietro Genovese, Luigi Naldini

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

精准基因编辑可在短暂的p53介导的DNA损伤反应后保留造血干细胞功能

Giulia Schiroli ,Anastasia Conti ,Samuele Ferrari ,Lucrezia Della Volpe ,Aurelien Jacob ,Luisa Albano ,Stefano Beretta ,Andrea Calabria ,Valentina Vavassori ,Patrizia Gasparini ,Eralda Salataj ,Delphine Ndiaye-Lobry ,Chiara Brombin ,Julie Chaumeil ,Eugenio Montini ,Ivan Merelli ,Pietro Genovese ,Luigi Naldini ,Raffaella Di Micco