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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
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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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