Edging closer to successful gene therapy for Wilson disease

被引：1
作者：
Sandahl, Thomas Damgaard [1 ]
Medici, Valentina [2 ]
机构：
[1] Aarhus Univ Hosp, Dept Hepatol & Gastroenterol, Aarhus, Denmark
[2] Univ Calif Davis, Div Gastroenterol & Hepatol, Dept Internal Med, Davis, CA 95616 USA
来源：
MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT | 2022年 / 27卷
关键词：
D O I：
10.1016/j.omtm.2022.10.005
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
引用
收藏
页码：293 / 294
页数：2
相关论文
共 5 条
  • [1] Wilson's disease
    Ala, Aftab
    Walker, Ann P.
    Ashkan, Keyoumars
    Dooley, James S.
    Schilsky, Michael L.
    [J]. LANCET, 2007, 369 (9559) : 397 - 408
  • [2] Long-term metabolic correction of Wilson's disease in a murine model by gene therapy
    Murillo, Oihana
    Moreno Luqui, Daniel
    Gazquez, Cristina
    Martinez-Espartosa, Debora
    Navarro-Blasco, Inigo
    Ignacio Monreal, Jose
    Guembe, Laura
    Moreno-Cermeno, Armando
    Corrales, Fernando J.
    Prieto, Jesus
    Hernandez-Alcoceba, Ruben
    Gonzalez-Aseguinolaza, Gloria
    [J]. JOURNAL OF HEPATOLOGY, 2016, 64 (02) : 419 - 426
  • [3] Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice
    Padula, Agnese
    Petruzzelli, Raffaella
    Philbert, Sasha A.
    Church, Stephanie J.
    Esposito, Federica
    Campione, Severo
    Monti, Marcello
    Capolongo, Filomena
    Perna, Claudia
    Nusco, Edoardo
    Schmidt, Hartmut H.
    Auricchio, Alberto
    Cooper, Garth J. S.
    Polishchuk, Roman
    Piccolo, Pasquale
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2022, 26 : 495 - 504
  • [4] Protein Splicing of Inteins: A Powerful Tool in Synthetic Biology
    Wang, Hao
    Wang, Lin
    Zhong, Baihua
    Dai, Zhuojun
    [J]. FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY, 2022, 10
  • [5] Bis-choline tetrathiomolybdate in patients with Wilson's disease: an open-label, multicentre, phase 2 study
    Weiss, Karl Heinz
    Askari, Frederick K.
    Czlonkowska, Anna
    Ferenci, Peter
    Bronstein, Jeff M.
    Bega, Danny
    Ala, Aftab
    Nicholl, David
    Flint, Susan
    Olsson, Lars
    Plitz, Thomas
    Bjartmar, Carl
    Schilsky, Michael L.
    [J]. LANCET GASTROENTEROLOGY & HEPATOLOGY, 2017, 2 (12) : 869 - 876
1