Therapeutic Levels of Anti-Sickling Hemoglobin Production in Erythroid Progeny of Sickle Cell Disease CD34+Cells Following Lentivirus Vector-Mediated Gene Delivery

被引:0
|
作者
Urbinati, Fabrizia [1 ]
Hargrove, Phillip W. [2 ]
Geiger, Sabine [1 ]
Romero, Zulema [1 ]
Wherley, Jennifer [1 ]
Kaufman, Michael L. [1 ]
Hollis, Roger P. [1 ]
Chambers, Christopher B. [3 ]
Persons, Derek A. [2 ]
Kohn, Donald B. [1 ]
Wilber, Andrew [3 ]
机构
[1] Univ Calif Los Angeles, Los Angeles, CA USA
[2] St Jude Childrens Res Hosp, Memphis, TN 38105 USA
[3] So Illinois Univ, Sch Med, Springfield, IL USA
关键词
D O I
暂无
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
26
引用
收藏
页码:S10 / S11
页数:2
相关论文
共 14 条
  • [1] Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells
    Urbinati, Fabrizia
    Hargrove, Phillip W.
    Geiger, Sabine
    Romero, Zulema
    Wherley, Jennifer
    Kaufman, Michael L.
    Hollis, Roger P.
    Chambers, Christopher B.
    Persons, Derek A.
    Kohn, Donald B.
    Wilber, Andrew
    EXPERIMENTAL HEMATOLOGY, 2015, 43 (05) : 346 - 351
  • [2] Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer
    Wilber, Andrew
    Hargrove, Phillip W.
    Kim, Yoon-Sang
    Riberdy, Janice M.
    Sankaran, Vijay G.
    Papanikolaou, Eleni
    Georgomanoli, Maria
    Anagnou, Nicholas P.
    Orkin, Stuart H.
    Nienhuis, Arthur W.
    Persons, Derek A.
    BLOOD, 2011, 117 (10) : 2817 - 2826
  • [3] Efficient lentivirus mediated gene transfer of an anti-sickling β-globin gene variant to CD34+ bone marrow cells from sickle cell disease patients
    Dalle, B
    Payen, E
    Pawliuk, R
    Westerman, K
    Beuzard, Y
    Leboulch, P
    MOLECULAR THERAPY, 2003, 7 (05) : S407 - S408
  • [4] Cyclosporin H Improves the Transduction of CD34+ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease
    Mormin, Mirella
    Rigonnot, Luc
    Chalumeau, Anne
    Miccio, Annarita
    Fournier, Clemence
    Pajanissamy, Sandya
    Dewannieux, Marie
    Galy, Anne
    HUMAN GENE THERAPY, 2024, 35 (21-22) : 896 - 903
  • [5] Efficient lentivirus mediated gene transfer of an anti-sickling beta-globin gene variant to CD34+bone marrow cells from sickle cell disease patients
    Payen, E
    Dalle, B
    Pawliuk, R
    Westerman, K
    Beuzard, Y
    Leboulch, P
    EXPERIMENTAL HEMATOLOGY, 2003, 31 (07) : 92 - 92
  • [6] Lentiviral Vector-Mediated Transfer of the Gamma-Globin Gene Into Normal and Beta-Thalassemic Human CD34+ Cells Results in Potentially Therapeutic Levels of Fetal Hemoglobin in Erythroid Progeny
    Wilber, Andrew
    Hargrove, Phillip W.
    Kim, Yoon-Sang
    Nienhuis, Arthur W.
    Persons, Derek A.
    BLOOD, 2009, 114 (22) : 1386 - 1387
  • [7] βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells
    Demirci, Selami
    Gudmundsdottir, Bjorg
    Li, Quan
    Haro-Mora, Juan J.
    Nassehi, Tina
    Drysdale, Claire
    Yapundich, Morgan
    Gamer, Jackson
    Seifuddin, Fayaz
    Tisdale, John F.
    Uchida, Naoya
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 : 912 - 921
  • [8] Development of an Objective Image-Based Flow Cytometry Sickling Assay for Quantification of Amelioration of Sickle Cell Disease Phenotype in Erythroid Differentiated CD34+Cells
    Lewis, Gretchen
    Nguyen, Brandon
    Pierciey, Francis J.
    Shestopalov, Ilya
    Veres, Gabor
    Bonner, Melissa
    MOLECULAR THERAPY, 2019, 27 (04) : 68 - 68
  • [9] Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+Cells Modified with a βAS3-Globin Lentiviral Vector
    Urbinati, Fabrizia
    Garcia, Zulema Romero
    Geiger, Sabine
    de Assin, Rafael Ruiz
    Kuftinec, Gabriela
    Hollis, Roger P.
    De Oliveira, Satiro N.
    Wherley, Jennifer
    Cooper, Aaron
    Townes, Tim M.
    Crooks, Gay M.
    Read, Elizabeth J.
    Walters, Mark C.
    Meiselman, Herbert J.
    Coates, Thomas D.
    Marder, Victor J.
    Kohn, Donald B.
    BLOOD, 2011, 118 (21) : 1348 - 1348
  • [10] Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+Cells
    Moran, Kevin
    Ling, Hui
    Lessard, Samuel
    Vieira, Benjamin
    Vu Hong
    Holmes, Michael C.
    Reik, Andreas
    Dang, Denny
    Gray, David
    Levasseur, Dana
    Rimmele, Pauline
    BLOOD, 2018, 132