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CLINICAL STUDY OF VALUE OF BONE MARROW TRANSPLANT FOR STORAGE DISEASES

CLINICAL STUDY OF VALUE OF BONE MARROW TRANSPLANT FOR STORAGE DISEASES
骨髓移植治疗储存性疾病价值的临床研究
批准号：
6113650
负责人：
NEENA KAPOOR
金额：
$3.56万
依托单位：
UNIVERSITY OF SOUTHERN CALIFORNIA
依托单位国家：
美国
项目类别：
财政年份：
1998
资助国家：
美国
项目状态：
已结题
起止时间：
1998-12-01 至 1999-11-30
来源：
https://reporter.nih.gov/project-details/6113650
关键词：
bone marrow transplantation central nervous system clinical research gene therapy human subject human therapy evaluation inborn metabolism disorder metabolism disorder chemotherapy

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中文摘要
翻译
同种异体骨髓移植已被用于治疗储存疾病的患者在过去的十年。在未来，自体骨髓移植与基因修饰的造血干细胞也将用于治疗这些疾病。然而，BMT对储存病移植患者中枢神经系统功能的影响仍存在争议。本研究旨在确定BMT对中枢神经系统功能的影响。假设同种异体BMT将改变中枢神经系统储存病的自然史。
英文摘要
Allogenic bone marrow transplantation has been used to treat patients with storage diseases for the last decade. In the future, autologous BMT with genetically modified hematopoietic stem cells will also be used to treat these diseases. Controversy, however, still exists as to the effects of BMT on the central nervous system function of patients transplanted for storage disease. This study is to determine the effects of BMT on CNS function. The hypothesis is that allogeneic BMT will modify the natural history of CNS storage diseases.
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