Core A - Vector Core

核心 A - 矢量核心

• 批准号: 8935365
• 负责人: JULIE JOHNSTON
• 金额: $ 15.77万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-05-15 至 2020-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8935365
• 关键词: Basic Science; Biological Assay; Clinical; Clinical Trials; Contracts; DNA; Dependovirus; Development; Disease; Equipment; Familial Hypercholesterolemia; Goals; Grant; Heart Diseases; Hematological Disease; Inherited; Intervention; Lung diseases; National Heart, Lung, and Blood Institute; Pennsylvania; Process; Production; Public Health; Quality Control; Research; Research Personnel; Research Project Grants; Resources; Services; Technology; Technology Transfer; Therapeutic Studies; Translations; Universities; Viral; adeno-associated viral vector; base; candidate selection; design; gene therapy; lecithin cholesterol acyltransferase deficiency; lipid disorder; manufacturing process; meetings; novel; pre-clinical; pre-clinical research; preclinical study; programs; translational study; vector

ABSTRACT Core A: Vector Core The University of Pennsylvania (Penn) Vector Core is a critical resource for investigators requiring viral-based vectors for preclinical and translational studies for the development of gene therapies for acquired and inherited disease. The main objective of the Core is to provide advanced vector technologies derived from adeno-associated viruses (AAV) in order to facilitate the translation of gene therapy research into clinical intervention. In support of this P01 entitled “AAV Gene Therapy for Lipid Disorders”, the Core will produce and quality control research and GMP process-comparable grade AAV vectors for preclinical and IND-enabling studies focused on treatment of familial lecithin-cholesterol acyltransferase (LCAT) deficiency and homozygous familial hypercholesterolemia (hoFH). The Core will also transfer the AAV vector manufacturing technology developed at Penn to a contract manufacturing organization (CMO) to support the proposed hoFH clinical trial, as well as develop new assays for extensive characterization of the clinical AAV vector product.

抽象的 核心A：向量核心 宾夕法尼亚大学（Penn）矢量核心是需要基于病毒的研究人员的关键资源 临床前和转化研究的向量，用于开发获得的基因疗法和 遗传疾病。核心的主要目的是提供从 腺相关病毒（AAV），以促进基因治疗研究转化为临床 干涉。为了支持这种标题为“脂质疾病的AAV基因疗法”的P01，核心将产生和 质量控制研究和GMP过程可相比之下的AAV级向量，用于临床前和成分 研究重点是治疗卵磷脂 - 胆固醇酰基转移酶（LCAT）缺乏症和纯合子 家族性高胆固醇血症（HOFH）。核心还将转移AAV矢量制造技术 在宾夕法尼亚州开发合同制造组织（CMO），以支持拟议的HOFH临床试验， 以及开发新的主张，以广泛表征临床AAV矢量产品。