Developing lentiviral vectors for gene therapy of Wiskott-Aldrich syndrome

被引：0
|
作者：
Evans-Galea, MV
Wielgosz, MM
Strom, TS
Hanawa, H
Cunningham, JM
Nienhuis, AW
机构：
[1] St Jude Childrens Res Hosp, Memphis, TN 38105 USA
[2] Memphis VA Med Ctr, Memphis, TN USA
[3] Nippon Med Coll, Tokyo 113, Japan
来源：
BLOOD | 2004年 / 104卷 / 11期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
3170
引用
收藏
页码：866A / 867A
页数：2
相关论文
共 50 条
  • [21] Gene therapy goes the distance in Wiskott-Aldrich syndrome
    Biffi, Alessandra
    NATURE MEDICINE, 2022, 28 (01) : 24 - 25
  • [22] Lentiviral gene therapy shows promising results for Wiskott-Aldrich syndrome patients (380 words)
    Patience, Jonny
    EXPERT REVIEW OF CLINICAL IMMUNOLOGY, 2013, 9 (08) : 704 - 704
  • [23] Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector
    Singh, Swati
    Khan, Iram
    Khim, Socheath
    Seymour, Brenda
    Sommer, Karen
    Wielgosz, Matthew
    Norgaard, Zachary
    Kiem, Hans-Peter
    Adair, Jennifer
    Liggitt, Denny
    Nienhuis, Arthur
    Rawlings, David J.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2017, 4 : 1 - 16
  • [24] Comparison of Insulators and Promoters for Expression of the Wiskott-Aldrich Syndrome Protein Using Lentiviral Vectors
    Koldej, Rachel M.
    Carney, Gael
    Wielgosz, Matthew M.
    Zhou, Sheng
    Zhan, Jun
    Sorrentino, Brian P.
    Nienhuis, Arthur W.
    HUMAN GENE THERAPY CLINICAL DEVELOPMENT, 2013, 24 (02) : 77 - 85
  • [25] Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome
    Ferrua, Francesca
    Cicalese, Maria Pia
    Galimberti, Stefania
    Scaramuzza, Samantha
    Giannelli, Stefania
    Pajno, Roberta
    Dionisio, Francesca
    Biasco, Luca
    Castiello, Maria Carmina
    Casiraghi, Miriam
    Facchini, Marcella
    Finocchi, Andrea
    Metin, Ayse
    Orange, Jordan S.
    Albert, Michael H.
    Petrescu, Carmen
    Bosticardo, Marita
    Villa, Anna
    Dott, Chris
    van Rossem, Koen
    Valsecchi, Maria Grazia
    Ciceri, Fabio
    Roncarolo, Maria Grazia
    Naldini, Luigi
    Aiuti, Alessandro
    BLOOD, 2015, 126 (23)
  • [26] Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy
    Wielgosz, Matthew M.
    Kim, Yoon-Sang
    Carney, Gael G.
    Zhan, Jun
    Reddivari, Muralidhar
    Coop, Terry
    Heath, Richard J.
    Brown, Scott A.
    Nienhuis, Arthur W.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2015, 2 : 14063
  • [27] Integration Site Analysis of Wiskott-Aldrich Syndrome Patients Treated with Lentiviral Vector Gene Therapy
    Biasco, Luca
    Baricordi, Cristina
    Dionisio, Francesca
    Calabria, Andrea
    Scaramuzza, Samantha
    Pellin, Danilo
    Di Serio, Clelia
    Montini, Eugenio
    von Kalle, Christof
    Schmidt, Manfred
    Aiuti, Alessandro
    MOLECULAR THERAPY, 2012, 20 : S86 - S86
  • [28] Evidence for efficacy and safety of lentiviral mediated gene transfer in Wiskott-Aldrich syndrome
    Roncarolo, Maria Grazia
    Bosticardo, Marita
    Marangoni, Francesco
    Scaramuzza, Samantha
    Trifari, Sara
    Galy, Anne
    Dupre, Loic
    Naldini, Luigi
    Aiuti, Alessandro
    Villa, Anna
    BLOOD CELLS MOLECULES AND DISEASES, 2008, 40 (02) : 282 - 282
  • [29] Lentiviral Gene Therapy Corrects Platelet Phenotype and Function in Wiskott-Aldrich Patients
    Sereni, Lucia
    Castiello, Maria Carmina
    Di Silvestre, Dario
    Della Valle, Patrizia
    Brombin, Chiara
    Ferrua, Francesca
    Cicalese, Maria Pia
    Pozzi, Loris
    Migliavacca, Maddalena
    Bernardo, Maria Ester
    Pignata, Claudia
    Farah, Roula
    Notarangelo, Lucia Dora
    Marcus, Nufar
    Cattaneo, Lorella
    Spinelli, Marco
    Giannelli, Stefania
    Bosticardo, Marita
    van Rossem, Koen
    D'Angelo, Armando
    Aiuti, Alessandro
    Mauri, Pierluigi
    JOURNAL OF CLINICAL IMMUNOLOGY, 2019, 39 : S80 - S81
  • [30] Wiskott-Aldrich Syndrome:: from molecular diagnosis to gene therapy
    Boztug, K
    Baumann, U
    Dewey, R
    Díez, IA
    Hansen, G
    Welte, K
    Klein, C
    KLINISCHE PADIATRIE, 2006, 218 (03): : 198 - 198
12345