Gene therapy goes the distance in Wiskott-Aldrich syndrome

被引：1
|
作者：
Biffi, Alessandra [1 ,2 ]
机构：
[1] Padua Univ Hosp, Pediat Hematol Oncol & Stem Cell Transplant Div, Padua, Italy
[2] Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, Boston, MA 02115 USA
来源：
NATURE MEDICINE | 2022年 / 28卷 / 01期
关键词：
EFFICACY;
D O I：
10.1038/s41591-021-01653-7
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Long-term follow-up data reinforce the curative potential of hematopoietic stem-cell gene therapy for this rare primary immunodeficiency disorder.
引用
收藏
页码：24 / 25
页数：2
相关论文
共 50 条
  • [1] Gene therapy goes the distance in Wiskott–Aldrich syndrome
    Alessandra Biffi
    [J]. Nature Medicine, 2022, 28 : 24 - 25
  • [2] Gene Therapy for Wiskott-Aldrich Syndrome
    Bosticardo, Marita
    Ferrua, Francesca
    Cavazzana, Marina
    Aiuti, Alessandro
    [J]. CURRENT GENE THERAPY, 2014, 14 (06) : 413 - 421
  • [3] Gene therapy of Wiskott-Aldrich syndrome
    Aiuti, A.
    Marangoni, F.
    Scaramuzza, S.
    Panaroni, C.
    Bosticardo, M.
    Trifari, S.
    Locci, M.
    Draghici, E.
    Galy, A.
    Naldini, L.
    Chiesa, R.
    Dupre, L.
    Villa, A.
    Roncarolo, M. G.
    [J]. HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2007, 92 (06): : 42 - 42
  • [4] Gene Therapy in Wiskott-Aldrich Syndrome
    Leiner, Peter
    [J]. ALLERGO JOURNAL, 2014, 23 (01) : 13 - 13
  • [5] Gene therapy for Wiskott-Aldrich Syndrome
    Aiuti, Alessandro
    [J]. HUMAN GENE THERAPY, 2014, 25 (11) : A6 - A7
  • [6] Gene therapy for Wiskott-Aldrich syndrome
    Witzel, Maximilian Georg Wolfgang
    Braun, Christian Joerg
    Boztug, Kaan
    Klein, Christoph
    [J]. EXPERT OPINION ON ORPHAN DRUGS, 2013, 1 (09): : 705 - 715
  • [7] Gene therapy for the Wiskott-Aldrich syndrome
    Galy, Anne
    Thrasher, Adrian J.
    [J]. CURRENT OPINION IN ALLERGY AND CLINICAL IMMUNOLOGY, 2011, 11 (06) : 545 - 550
  • [8] Gene Edition for Wiskott-Aldrich Syndrome Gene Therapy
    Martin, Francisco
    Munoz, Pilar
    Cobo, Marien
    Sanchez-Gilabert, Almudena
    Real, Pedro
    Ramos, Veronica
    Gregory, Philip D.
    Holmes, Michael C.
    Toscano, Miguel G.
    [J]. MOLECULAR THERAPY, 2015, 23 : S136 - S136
  • [9] Gene therapy in Wiskott-Aldrich syndrome: The next step?
    Julie Y. Patel
    David P. Huston
    [J]. Current Allergy and Asthma Reports, 2005, 5 (5) : 347 - 348
  • [10] Gene therapy approaches for the Wiskott-Aldrich syndrome.
    [J]. BLOOD CELLS MOLECULES AND DISEASES, 2002, 28 (03) : 326 - 327
12345