Gene therapy of murine GM1 gangliosidosis by genetically modified bone marrow hematopoietic progenitor cells.

被引：0
|
作者：
Sano, R [1 ]
Tessitore, A [1 ]
Mann, L [1 ]
d'Azzo, A [1 ]
机构：
[1] St Jude Childrens Res Hosp, Memphis, TN 38105 USA
来源：
AMERICAN JOURNAL OF HUMAN GENETICS | 2003年 / 73卷 / 05期
关键词：
D O I：
暂无
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
2693
引用
收藏
页码：627 / 627
页数：1
相关论文
共 50 条
  • [21] Application of a pentasaccharide biomarker to access treatment efficacy of gene therapy for GM1 gangliosidosis
    Kell, Pamela
    Mishra, Sonali
    D'Souza, Precilla
    Tifft, Cynthia J.
    Jiang, Xuntian
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [22] Retroviral-mediated transduction of the green fluorescent protein gene into murine bone marrow allows stringent purification and selective transplantation of genetically modified pluripotent hematopoietic cells.
    Persons, DA
    Allay, JA
    Riberdy, JM
    Nienhuis, AW
    BLOOD, 1997, 90 (10) : 515 - 515
  • [23] BONE-MARROW FINDINGS IN HETEROZYGOTES FOR OPHTHALMOPLEGIC NEUROLIPIDOSIS AND GM1 GANGLIOSIDOSIS TYPE-2
    BOLTSHAUSER, E
    ADANK, S
    BRANDLE, B
    GALLINO, L
    NEUROPEDIATRICS, 1987, 18 (02) : 118 - 118
  • [24] Spatiotemporal expression of GM1 in murine medial pallial neural progenitor cells
    Liour, SS
    Dinkins, MB
    Su, CY
    Yu, RK
    JOURNAL OF COMPARATIVE NEUROLOGY, 2005, 491 (04) : 330 - 338
  • [25] Spatiotemporal expression of GM1 in murine medial pallial neural progenitor cells
    Yu, RK
    Dinkins, MB
    Su, CY
    Liour, SS
    JOURNAL OF NEUROCHEMISTRY, 2005, 94 : 22 - 22
  • [26] Correlation between adhesion molecule expression and homing to bone marrow by murine fetal liver hematopoietic progenitor cells.
    Wolber, FM
    Yoder, MC
    Leonard, E
    Michael, S
    Orschell-Traycoff, C
    Srour, EF
    BLOOD, 2000, 96 (11) : 685A - 685A
  • [27] Hematopoietic stem cell gene therapy corrects lysosomal storage in CNS in murine model of GM1-gangliosidosis
    Tsunogai, T.
    Ohashi, T.
    Shimada, Y.
    Higuchi, T.
    Kimura, A.
    Matsushima, S.
    Iizuka, S.
    Watabe, A.
    Kato, F.
    Kobayashi, H.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A101 - A101
  • [28] Comparing GM1 gangliosidosis patients treated and untreated with gene therapy: Longitudinal white matter changes in GM1 using differential tractography
    Acosta, Maria T.
    Lewis, Connor J.
    Vardar, Zeynep
    Johnston, Jean M.
    Kuhn, Anna
    D'Souza, Precilla
    Shazeeb, Mohammed Salman
    Tifft, Cynthia J.
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [29] Role of proteoglycans in the interaction between hematopoietic progenitor cells and bone marrow endothelial cells.
    Netelenbos, T
    Huijgens, PC
    van Haperen, MJAM
    Hof, BJV
    van Dijk, W
    Dräger, AM
    EXPERIMENTAL HEMATOLOGY, 1999, 27 (07) : 42 - 42
  • [30] Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis
    Gross, Amanda L.
    Gray-Edwards, Heather L.
    Bebout, Cassie N.
    Ta, Nathan L.
    Nielsen, Kayly
    Brunson, Brandon L.
    Mercado, Kalajan R. Lopez
    Osterhoudt, Devin E.
    Batista, Ana Rita
    Maitland, Stacy
    Seyfried, Thomas N.
    Sena-Esteves, Miguel
    Martin, Douglas R.
    BRAIN, 2022, 145 (02) : 655 - 669
12345