Long-Term Effects of Hematopoietic Stem Cell Gene Therapy in the Murine Model of Wiskott-Aldrich Syndrome: Persistence of Functional Correction of T Cells and Lack of Malignant Transformation

被引：0
|
作者：
Marangoni, Francesco [1 ,2 ]
Dupre, Loic [1 ]
Scaramuzza, Samantha [1 ]
Panaroni, Cristina [1 ]
Trifari, Sara [1 ]
Hernandez, Raisa Jofra [1 ]
Thrasher, Adrian J. [3 ]
Galy, Anne [4 ]
Aiuti, Alessandro [1 ]
Naldini, Luigi [1 ,2 ]
Roncarolo, Maria Grazia [1 ,2 ]
机构：
[1] Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy HSR TIGET, I-20132 Milan, Italy
[2] Univ Vita Salute San Raffaele, San Raffaele Sci Inst, Milan, Italy
[3] Inst Child Hlth, Mol Immunol Unit, London, England
[4] CNRS, UMR 8115, Evry, France
来源：
MOLECULAR THERAPY | 2006年 / 13卷
关键词：
D O I：
10.1016/j.ymthe.2006.08.081
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
65
引用
收藏
页码：S27 / S28
页数：2
相关论文
共 50 条
  • [21] Characterization of the genomic insertion of a lentiviral vector developed for the gene therapy of Wiskott-Aldrich syndrome, in murine hematopoietic cells
    Mantovani, Julie
    Charrier, Sabine
    Danos, Olivier
    Perea, Javier
    Galy, Anne
    HUMAN GENE THERAPY, 2007, 18 (10) : 1012 - 1012
  • [22] Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome
    Magnani, Alessandra
    Semeraro, Michaela
    Adam, Frederic
    Booth, Claire
    Dupre, Loic
    Morris, Emma C.
    Gabrion, Aurelie
    Roudaut, Cecile
    Borgel, Delphine
    Toubert, Antoine
    Clave, Emmanuel
    Abdo, Chrystelle
    Gorochov, Guy
    Petermann, Rachel
    Guiot, Melanie
    Miyara, Makoto
    Moshous, Despina
    Magrin, Elisa
    Denis, Adeline
    Suarez, Felipe
    Lagresle-Peyrou, Chantal
    Doto, Aoife
    Everett, John K.
    Trinquand, Amelie
    Guisset, Marianne
    Xu-Bayford, Jin Hua
    Hacein-Bey-Abina, Salima
    Kauskot, Alexandre
    Elfeky, Reem
    Rivat, Christine
    Abbas, Sarah
    Gaspar, Bobby H.
    Macintyre, Elizabeth A.
    Picard, Capucine
    Bushman, Frederic D.
    Galy, Anne
    Fischer, Alain
    Six, Emmanuelle
    Thrasher, Adrian J.
    Cavazzana, Marina
    BLOOD, 2021, 138
  • [23] B cell reconstitution in Wiskott-Aldrich Syndrome murine model upon lentiviral-mediated gene therapy
    Bosticardo, M.
    Draghici, E.
    Schena, F.
    Locci, M.
    Scaramuzza, S.
    Marangoni, F.
    Traggiai, E.
    Galy, A.
    Naldini, L.
    Aiuti, A.
    Villa, A.
    Roncarolo, M. G.
    CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 2008, 154 : 96 - 97
  • [24] LONG-TERM ALLOSPECIFIC T-CELL LINES AS A TOOL TO DEFINE DEFECTS IN THE WISKOTT-ALDRICH SYNDROME
    MOLINA, IJ
    KENNEY, DM
    ROSEN, FS
    REMOLDODONNELL, E
    FASEB JOURNAL, 1992, 6 (04): : A1120 - A1120
  • [25] CUTANEOUS T-CELL LYMPHOMA AND LEUKOCYTOCLASTIC VASCULITIS IN A LONG-TERM SURVIVOR OF WISKOTT-ALDRICH SYNDROME
    WATSON, RD
    GERSHWIN, ME
    SMITHWICK, E
    CASTLES, JJ
    RUEBNER, B
    ANNALS OF ALLERGY, 1985, 55 (05): : 654 - &
  • [26] Lentiviral-Mediated Gene Therapy Leads to B Cell Reconstitution in Wiskott-Aldrich Syndrome Murine Model
    Bosticardo, Marita
    Draghici, Elena
    Schena, Francesca
    Sauer, Aisha
    Locci, Michela
    Scaramuzza, Samantha
    Marangoni, Francesco
    Traggiai, Elisabetta
    Galy, Anne
    Naldini, Luigi
    Aiuti, Alessandro
    Roncarolo, Maria Grazia
    Villa, Anna
    MOLECULAR THERAPY, 2009, 17 : S375 - S375
  • [27] Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice
    Klein, C
    Nguyen, D
    Liu, CH
    Mizoguchi, A
    Bhan, AK
    Miki, H
    Takenawa, T
    Rosen, FS
    Alt, FW
    Mulligan, RC
    Snapper, SB
    BLOOD, 2003, 101 (06) : 2159 - 2166
  • [28] Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome
    Catucci, M.
    Prete, F.
    Bosticardo, M.
    Castiello, M. C.
    Draghici, E.
    Locci, M.
    Roncarolo, M. G.
    Aiuti, A.
    Benvenuti, F.
    Villa, A.
    GENE THERAPY, 2012, 19 (12) : 1150 - 1158
  • [29] Long-term outcome and chimerism in patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation: a retrospective nationwide survey
    Iguchi, Akihiro
    Cho, Yuko
    Yabe, Hiromasa
    Kato, Shunichi
    Kato, Koji
    Hara, Junichi
    Koh, Katsuyoshi
    Takita, Junko
    Ishihara, Takashi
    Inoue, Masami
    Mai, Kohsuke
    Nakayama, Hideki
    Hashii, Yoshiko
    Morimoto, Akira
    Atsuta, Yoshiko
    Morio, Tomohiro
    INTERNATIONAL JOURNAL OF HEMATOLOGY, 2019, 110 (03) : 364 - 369
  • [30] Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome (Aug, 10.1038/s41591-021-01641-x, 2022)
    Magnani, A.
    Semeraro, M.
    Adam, F.
    Booth, C.
    Dupre, L.
    Morris, E. C.
    Gabrion, A.
    Roudaut, C.
    Borgel, D.
    Toubert, A.
    Clave, E.
    Abdo, C.
    Gorochov, G.
    Petermann, R.
    Guiot, M.
    Miyara, M.
    Moshous, D.
    Magrin, E.
    Denis, A.
    Suarez, F.
    Lagresle, C.
    Roche, A. M.
    Everett, J.
    Trinquand, A.
    Guisset, M.
    Bayford, J. Xu
    Hacein-Bey-Abina, S.
    Kauskot, A.
    Elfeky, R.
    Rivat, C.
    Abbas, S.
    Gaspar, H. B.
    Macintyre, E.
    Picard, C.
    Bushman, F. D.
    Galy, A.
    Fischer, A.
    Six, E.
    Thrasher, A. J.
    Cavazzana, M.
    NATURE MEDICINE, 2022, 28 (10) : 2217 - 2217
12345