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Models and Gene Therapies for AAT Deficiency

Models and Gene Therapies for AAT Deficiency
AAT 缺乏症的模型和基因疗法
批准号：
10463803
负责人：
Terence R. Flotte
金额：
$8.38万
依托单位：
UNIV OF MASSACHUSETTS MED SCH WORCESTER
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
项目状态：
未结题
起止时间：
2021-08-09 至 2026-07-31
来源：
https://reporter.nih.gov/project-details/10463803
关键词：
AccountingBudgetsCommunicationCommunication ResearchComplexConsultationsEnsureFacultyFeedbackGoalsGrantHuman ResourcesInstitutesIowaMassachusettsModelingOccupational activity of managing financesPersonsProgress ReportsResearchResearch PersonnelScheduleScientistStructureTimeUniversitiesWorkalpha 1-Antitrypsin Deficiencydata exchangegene therapymedical schoolsmeetingsmemberprogramstherapeutic RNAvirtual

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中文摘要
翻译
行政核心 项目摘要 行政核心将提供一个结构，以管理本组织的财务方面。 复杂的多组件程序，并创建车辆之间的通信 调查人员负责每个组成部分。行政核心还将召集内部和 和外部咨询委员会成员，作为客观但知情的科学家， 向P01研究者反馈每个组成部分的优缺点 并评估每个组成部分（以及整个计划）随时间的进展情况。
英文摘要
Administrative Core Project Summary The administrative core will provide a structure for management of the financial aspects of this complex multi-component program and create vehicle for communication among the investigators leading each component. The administrative core will also convene both internal and external advisory board members, as objective but informed scientists to provide feedback to the P01 investigators about the strengths and weaknesses of each component and to evaluate the progress of each component (and of the overall program) over time.
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Models and Gene Therapies for AAT Deficiency
Models and Gene Therapies for AAT Deficiency
Optimized Gene Replacement for AAT deficiency and Modeling of Clinical Outcomes in small and large animal models
Optimized Gene Replacement for AAT deficiency and Modeling of Clinical Outcomes in small and large animal models
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