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TREATING SCID DUE TO ADA DEFICIENCY WITH CD34+ SELECTED AUTOLOGOUS STEM CELLS

TREATING SCID DUE TO ADA DEFICIENCY WITH CD34+ SELECTED AUTOLOGOUS STEM CELLS
使用 CD34 精选自体干细胞治疗因 ADA 缺乏所致的 SCID
批准号：
6308615
负责人：
Diane W. Wara
金额：
$2.58万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
依托单位国家：
美国
项目类别：
财政年份：
1999
资助国家：
美国
项目状态：
已结题
起止时间：
1999-12-01 至 2000-11-30
来源：
https://reporter.nih.gov/project-details/6308615
关键词：
CD34 molecule adenosine deaminase autologous transplantation cell transplantation clinical research enzyme deficiency gene therapy genetic transduction hematopoietic stem cells hematopoietic tissue transplantation human genetic material tag human subject human therapy evaluation newborn human (0-6 weeks) severe combined immunodeficiency

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中文摘要
翻译
本研究将验证一种假设，即通过逆转录病毒介导的基因转移将正常人ADA互补DNA转导的CD34+脐带血干细胞自体移植后，腺苷脱氨酶（ADA）缺陷严重联合免疫缺陷综合征（SCID）患者的免疫功能重建。该方案的具体目的是：1)确定转导的CD34+脐带血干细胞是否会在ADA缺乏症婴儿自体移植后植入；2)确定在不同的造血细胞群中植入的程度和持久性；3)确定基因治疗后免疫重建的程度和持久性。
英文摘要
This study will test the hypothesis that reconstitution of immune function in adenosine deaminase (ADA)-deficient severe combined immunodeficiency syndrome (SCID) will result from the autologous transplantation of CD34+ umbilical cord blood stem cells that have been transduced with a normal human ADA complementary DNA by retroviral-mediated gene transfer. The specific aims of this protocol are to: 1)determine whether transduced CD34+ umbilical cord blood stem cells will engraft following autologous transplantation in an infant with ADA deficiency; 2) determine the extent and durability of engraftment in different hematopoietic cell populations; 3) determine the extent and durability of immune reconstitution following gene therapy.
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