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

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

造血干细胞基因治疗Wiskott-Aldrich综合征的疗效

Labrosse, Roxane; Chu, Julia I; Armant, Myriam A; Everett, John K; Pellin, Danilo; Kareddy, Niharika; Frelinger, Andrew L; Henderson, Lauren A; O'Connell, Amy E; Biswas, Amlan; Coenen-van der Spek, Jet; Miggelbrink, Alexandra; Fiorini, Claudia; Adhikari, Hriju; Berry, Charles C; Cantu, Vito Adrian; Fong, Johnson; Jaroslavsky, Jason; Karadeniz, Derin F; Li, Quan-Zhen; Reddy, Shantan; Roche, Aoife M; Zhu, Chengsong; Whangbo, Jennifer S; Dansereau, Colleen; Mackinnon, Brenda; Morris, Emily; Koo, Stephanie M; London, Wendy B; Baris, Safa; Ozen, Ahmet; Karakoc-Aydiner, Elif; Despotovic, Jenny M; Forbes Satter, Lisa R; Saitoh, Akihiko; Aizawa, Yuta; King, Alejandra; Nguyen, Mai Anh Thi; Vu, Vy Do Uyen; Snapper, Scott B; Galy, Anne; Notarangelo, Luigi D; Bushman, Frederic D; Williams, David A; Pai, Sung-Yun

RNA polymerase II pausing temporally coordinates cell cycle progression and erythroid differentiation.

RNA聚合酶II的暂停在时间上协调细胞周期进程和红系分化

Martell Danya J, Merens Hope E, Caulier Alexis, Fiorini Claudia, Ulirsch Jacob C, Ietswaart Robert, Choquet Karine, Graziadei Giovanna, Brancaleoni Valentina, Cappellini Maria Domenica, Scott Caroline, Roberts Nigel, Proven Melanie, Roy Noémi B A, Babbs Christian, Higgs Douglas R, Sankaran Vijay G, Churchman L Stirling

Congenital anemia reveals distinct targeting mechanisms for master transcription factor GATA1

先天性贫血揭示了主转录因子 GATA1 的独特靶向机制

Ludwig, Leif S; Lareau, Caleb A; Bao, Erik L; Liu, Nan; Utsugisawa, Taiju; Tseng, Alex M; Myers, Samuel A; Verboon, Jeffrey M; Ulirsch, Jacob C; Luo, Wendy; Muus, Christoph; Fiorini, Claudia; Olive, Meagan E; Vockley, Christopher M; Munschauer, Mathias; Hunter, Abigail; Ogura, Hiromi; Yamamoto, Toshiyuki; Inada, Hiroko; Nakagawa, Shinichiro; Ohzono, Shuichi; Subramanian, Vidya; Chiarle, Roberto; Glader, Bertil; Carr, Steven A; Aryee, Martin J; Kundaje, Anshul; Orkin, Stuart H; Regev, Aviv; McCavit, Timothy L; Kanno, Hitoshi; Sankaran, Vijay G

Loss of FGFR4 promotes the malignant phenotype of PDAC

FGFR4 的缺失促进了 PDAC 的恶性表型。

D'Agosto, Sabrina; Pezzini, Francesco; Veghini, Lisa; Delfino, Pietro; Fiorini, Claudia; Temgue Tane, Gael D; Del Curatolo, Anais; Vicentini, Caterina; Ferrari, Giorgia; Pasini, Davide; Andreani, Silvia; Lupo, Francesca; Fiorini, Elena; Lorenzon, Giulia; Lawlor, Rita T; Rusev, Borislav; Malinova, Antonia; Luchini, Claudio; Milella, Michele; Sereni, Elisabetta; Pea, Antonio; Bassi, Claudio; Bailey, Peter; Scarpa, Aldo; Bria, Emilio; Corbo, Vincenzo

JAK/Stat5-mediated subtype-specific lymphocyte antigen 6 complex, locus G6D (LY6G6D) expression drives mismatch repair proficient colorectal cancer

JAK/Stat5介导的亚型特异性淋巴细胞抗原6复合物,G6D位点(LY6G6D)的表达驱动错配修复功能正常的结直肠癌

Giordano, Guido; Parcesepe, Pietro; D'Andrea, Mario Rosario; Coppola, Luigi; Di Raimo, Tania; Remo, Andrea; Manfrin, Erminia; Fiorini, Claudia; Scarpa, Aldo; Amoreo, Carla Azzurra; Conciatori, Fabiana; Milella, Michele; Caruso, Francesca Pia; Cerulo, Luigi; Porras, Almudena; Pancione, Massimo

Macrothrombocytopenia associated with a rare GFI1B missense variant confounding the presentation of immune thrombocytopenia

罕见的GFI1B错义变异引起的巨血小板减少症混淆了免疫性血小板减少症的临床表现

Cheng, Aaron N; Bao, Erik L; Fiorini, Claudia; Sankaran, Vijay G

Developmentally-faithful and effective human erythropoiesis in immunodeficient and Kit mutant mice

在免疫缺陷和Kit突变小鼠中实现发育忠实且有效的红细胞生成

Fiorini, Claudia; Abdulhay, Nour J; McFarland, Sean K; Munschauer, Mathias; Ulirsch, Jacob C; Chiarle, Roberto; Sankaran, Vijay G

Targeted Application of Human Genetic Variation Can Improve Red Blood Cell Production from Stem Cells

有针对性地应用人类基因变异可以提高干细胞造血红细胞的效率

Giani, Felix C; Fiorini, Claudia; Wakabayashi, Aoi; Ludwig, Leif S; Salem, Rany M; Jobaliya, Chintan D; Regan, Stephanie N; Ulirsch, Jacob C; Liang, Ge; Steinberg-Shemer, Orna; Guo, Michael H; Esko, Tõnu; Tong, Wei; Brugnara, Carlo; Hirschhorn, Joel N; Weiss, Mitchell J; Zon, Leonard I; Chou, Stella T; French, Deborah L; Musunuru, Kiran; Sankaran, Vijay G

UCP2, a mitochondrial protein regulated at multiple levels

UCP2是一种线粒体蛋白,其表达受多层次调控。

Donadelli, Massimo; Dando, Ilaria; Fiorini, Claudia; Palmieri, Marta