Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

被引：65
|
作者：
Chinen, Javier
Davis, Joie
De Ravin, Suk See
Hay, Beverly N.
Hsu, Amy P.
Linton, Gilda F.
Naumann, Nora
Nomicos, Effie Y. H.
Silvin, Christopher
Ulrick, Jean
Whiting-Theobald, Narda L.
Malech, Harry L.
Puck, Jennifer M.
机构：
[1] NIAID, Host Def Lab, NIH, Dept Hlth & Human Serv, Bethesda, MD 20892 USA
[2] NHGRI, Genet & Mol Biol Branch, NIAID, NIH,DHHS, Bethesda, MD 20892 USA
来源：
BLOOD | 2007年 / 110卷 / 01期
关键词：
BONE-MARROW-TRANSPLANTATION; CHRONIC GRANULOMATOUS-DISEASE; STEM-CELL TRANSPLANTATION; T-CELL; CD34(+) CELLS; TRANSDUCTION; SCID-X1; SURVIVAL; MODEL; IL2RG;
D O I：
10.1182/blood-2006-11-058933
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gamma c) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoletic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.
引用
收藏
页码：67 / 73
页数：7
相关论文
共 50 条
  • [41] Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy
    Bradford, Kathryn L.
    Liu, Siyu
    Krajinovic, Maja
    Ansari, Marc
    Garabedian, Elizabeth
    Tse, John
    Wang, Xiaoyan
    Shaw, Kit L.
    Gaspar, H. Bobby
    Candotti, Fabio
    Kohn, Donald B.
    BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2020, 26 (10) : 1819 - 1827
  • [42] Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency
    Reinhardt, Bryanna
    Habib, Omar
    Shaw, Kit L.
    Garabedian, Elizabeth
    Carbonaro-Sarracino, Denise A.
    Terrazas, Dayna
    Fernandez, Beatriz Campo
    De Oliveira, Satiro
    Moore, Theodore B.
    Ikeda, Alan K.
    Engel, Barbara C.
    Podsakoff, Gregory M.
    Hollis, Roger P.
    Fernandes, Augustine
    Jackson, Connie
    Shupien, Sally
    Mishra, Suparna
    Davila, Alejandra
    Mottahedeh, Jack
    Vitomirov, Andrej
    Meng, Wenzhao
    Rosenfeld, Aaron M.
    Roche, Aoife M.
    Hokama, Pascha
    Reddy, Shantan
    Everett, John
    Wang, Xiaoyan
    Prak, Eline T. Luning
    Cornetta, Kenneth
    Hershfield, Michael S.
    Sokolic, Robert
    De Ravin, Suk See
    Malech, Harry L.
    Bushman, Frederic D.
    Candotti, Fabio
    Kohn, Donald B.
    BLOOD, 2021, 138 (15) : 1304 - 1316
  • [43] Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X-Linked Adrenoleukodystrophy
    Cartier, Nathalie
    Aubourg, Patrick
    BRAIN PATHOLOGY, 2010, 20 (04) : 857 - 862
  • [44] Bone marrow transplantation as treatment for X-linked immunodeficiency with hyper-IgM
    Bordigoni, P
    Auburtin, B
    Carret, AS
    Schuhmacher, A
    Humbert, JC
    Le Deist, F
    Sommelet, D
    BONE MARROW TRANSPLANTATION, 1998, 22 (11) : 1111 - 1114
  • [45] New insights into the pathogenesis of adenosine deaminase-severe combined immunodeficiency and progress in gene therapy
    Sauer, Aisha V.
    Aiuti, Alessandro
    CURRENT OPINION IN ALLERGY AND CLINICAL IMMUNOLOGY, 2009, 9 (06) : 496 - 502
  • [46] Bone marrow transplantation as treatment for X-linked immunodeficiency with hyper-IgM
    P Bordigoni
    B Auburtin
    A-S Carret
    A Schuhmacher
    J-C Humbert
    F Le Deist
    D Sommelet
    Bone Marrow Transplantation, 1998, 22 : 1111 - 1114
  • [47] Bone marrow transplant for X-linked protoporphyria with severe hepatic fibrosis
    Butler, David F.
    Ginn, Kevin F.
    Daniel, James F.
    Bloomer, Joseph R.
    Kats, Alexander
    Shreve, Nancy
    Myers, Gary D.
    PEDIATRIC TRANSPLANTATION, 2015, 19 (04) : E106 - E110
  • [48] A major X-linked locus affects kidney function in mice
    Leduc, Magalie S.
    Savage, Holly S.
    Stearns, Timothy M.
    Cario, Clinton L.
    Walsh, Kenneth A.
    Paigen, Beverly
    Berndt, Annerose
    MOLECULAR GENETICS AND GENOMICS, 2012, 287 (11-12) : 845 - 854
  • [49] Severe X-linked chronic granulomatous disease in two unrelated females
    Chollet-Martin, Sylvie
    Lopez, Anne
    Gaud, Catherine
    Henry, Dominique
    Stos, Bertrand
    El Benna, Jamel
    Chedevile, Gaelle
    Gendrel, Dominique
    Gougerot-Pocidalo, Marie-Anne
    Grandchamp, Bernard
    Gerard, Benedicte
    EUROPEAN JOURNAL OF PEDIATRICS, 2007, 166 (02) : 153 - 159
  • [50] Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency
    Sokolic, Robert
    Candotti, Fabio
    EXPERT OPINION ON ORPHAN DRUGS, 2017, 5 (06): : 477 - 485
12345