靶向 BCL11A 的基因疗法可诱导胎儿血红蛋白并降低镰状细胞病患者的镰状血红蛋白-猫眼课题宝

靶向 BCL11A 的基因疗法可诱导胎儿血红蛋白并降低镰状细胞病患者的镰状血红蛋白

批准号：

10083551

负责人：

DAVID A WILLIAMS

金额：

$120.04万

依托单位：

BOSTON CHILDREN'S HOSPITAL

依托单位国家：

美国

项目类别：

财政年份：

2020

资助国家：

美国

项目状态：

已结题

起止时间：

2020-07-22 至 2024-12-31

来源：

https://reporter.nih.gov/project-details/10083551

关键词：

Adult Allogenic Architecture Attenuated Autologous Biocompatible Materials Biological Cells Clinical Data Disease Engraftment Enrollment Erythrocytes Erythroid Erythroid Cells Feasibility Studies Fetal Hemoglobin Fetal Reduction Future Gene Transfer Gene-Modified Genes Globin Goals Half-Life Health Hematology Hematopoietic Stem Cell Transplantation Hematopoietic stem cells Hemoglobin Hemoglobinopathies Human Incidence Individual Laboratories Lead Life MicroRNAs Molecular Outcome Patients Phenotype Risk Severities Siblings Sickle Cell Sickle Cell Anemia Sickle Hemoglobin Transgenes Transplantation base beta Thalassemia clinical effect cohort curative treatments disease phenotype fetal gamma Globin gene therapy gene transfer vector graft failure graft vs host disease improved in vivo knock-down mathematical model mortality mouse model mutant phase I trial polymerization pre-clinical prevent safety and feasibility sickling small hairpin RNA stem cells targeted treatment vector

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期刊论文(4)

专著(0)

科研奖励(0)

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Corrigendum.

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期刊：

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[]

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期刊：

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影响因子： 16.6

作者：

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通讯作者： Higgins, John M.