STEM CELL GENE THERAPY USING LONG TERM BONE MARROW CULTURE TRANSDUCTION PROTOCOL

使用长期骨髓培养转导方案的干细胞基因治疗

基本信息

  • 批准号:
    6591288
  • 负责人:
  • 金额:
    $ 11.11万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2002
  • 资助国家:
    美国
  • 起止时间:
    2002-05-01 至 2003-04-30
  • 项目状态:
    已结题

项目摘要

Attempts to introduce genes into hematopoietic stem cells (HSCs) have been disappointing In general, using murine retroviral based vectors to transduce CD34+ cells in vitro have resulted in a low level (0 1-1%) of gene marking for a limited period of time Studies in canines demonstrated that LTBMC transduced with retroviral vectors gave higher levels of gene marking in vivo The advantage of this technique is that by using unfractionated marrow there is no preselection; stem cells and retrovirus are able to colocalize on the stroma; the system selects for LTCIC; transduction occurs 3 times over 21 days following feeding so that cells are in cycle, improving transduction efficiency We examined the utility of this technique in rhesus macaques In 2 animals that underwent this autologous transplantation procedure, no conditioning regime was implemented Unfractionated marrow was transduced for 15 - 18 days with a PA317LN based vector Animals were monitored f or "neo" the marker gene in peripheral blood and bone marrow We observed between 1% and 3% gene marking in peripheral blood for approximately 3 months The frequency of neo in bone marrow was generally lower than that in peripheral blood This technique appears encouraging as our data are similar to what has been observed by others, but our protocol is in the absence of any conditioning regimen Recent efforts have focused on attempts to use LTBMC to transduce HSC using a retroviral vector encoding GFP Initial experiments using a retroviral vector provided by R Howley resulted in a failure to maintain LTBMC, which appears to be due to soluble factors provided by the retroviral producer cell line These results suggest that the LTBMC technique may be difficult to generalize to other retroviral vectors Results of these studies may prove useful in efforts to optimize genetic modification of human hematopoietic stem cells
将基因导入造血干细胞(hsc)的尝试

项目成果

期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)

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M ROSENZWEIG其他文献

M ROSENZWEIG的其他文献

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{{ truncateString('M ROSENZWEIG', 18)}}的其他基金

ESTABLISHMENT OF RHESUS BONE MARROW CHIMERAS
恒河猴骨髓嵌合体的建立
  • 批准号:
    6591322
  • 财政年份:
    2002
  • 资助金额:
    $ 11.11万
  • 项目类别:
IN VIVO ANALYSIS OF T CELL TURNOVER IN RHESUS MACAQUES
恒河猴 T 细胞更新的体内分析
  • 批准号:
    6591317
  • 财政年份:
    2002
  • 资助金额:
    $ 11.11万
  • 项目类别:
EXPRESSION OF BCL 2 IN SIV INFECTED THYMIC TISSUE
BCL 2 在 SIV 感染的胸腺组织中的表达
  • 批准号:
    6591316
  • 财政年份:
    2002
  • 资助金额:
    $ 11.11万
  • 项目类别:
FLT LIGAND PERIPHERAL BLOOD STEM CELL MOBILIZATION FOR STEM CELL GENE THERAPY
用于干细胞基因治疗的 FLT 配体外周血干细胞动员
  • 批准号:
    6591324
  • 财政年份:
    2002
  • 资助金额:
    $ 11.11万
  • 项目类别:
XENOGENEIC THYMIC TRANSPLANTATION AS AN ADJUNCT TO TREATMENT OF AIDS
异种胸腺移植作为艾滋病治疗的辅助手段
  • 批准号:
    6591306
  • 财政年份:
    2002
  • 资助金额:
    $ 11.11万
  • 项目类别:
EXPRESSION OF BCL 2 IN SIV INFECTED THYMIC TISSUE
BCL 2 在 SIV 感染的胸腺组织中的表达
  • 批准号:
    6453762
  • 财政年份:
    2001
  • 资助金额:
    $ 11.11万
  • 项目类别:
IN VIVO ANALYSIS OF T CELL TURNOVER IN RHESUS MACAQUES
恒河猴 T 细胞更新的体内分析
  • 批准号:
    6453763
  • 财政年份:
    2001
  • 资助金额:
    $ 11.11万
  • 项目类别:
STEM CELL GENE THERAPY USING LONG TERM BONE MARROW CULTURE TRANSDUCTION PROTOCOL
使用长期骨髓培养转导方案的干细胞基因治疗
  • 批准号:
    6453734
  • 财政年份:
    2001
  • 资助金额:
    $ 11.11万
  • 项目类别:
ESTABLISHMENT OF RHESUS BONE MARROW CHIMERAS
恒河猴骨髓嵌合体的建立
  • 批准号:
    6453768
  • 财政年份:
    2001
  • 资助金额:
    $ 11.11万
  • 项目类别:
FLT LIGAND PERIPHERAL BLOOD STEM CELL MOBILIZATION FOR STEM CELL GENE THERAPY
用于干细胞基因治疗的 FLT 配体外周血干细胞动员
  • 批准号:
    6453770
  • 财政年份:
    2001
  • 资助金额:
    $ 11.11万
  • 项目类别:

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