Core A - Vector Core

摘要 核心A：矢量核心 宾夕法尼亚大学（Penn）Vector Core是需要基于病毒的研究人员的关键资源。 用于临床前和转化研究的载体，用于开发获得性和 遗传性疾病核心的主要目标是提供先进的矢量技术， 腺相关病毒（AAV），以促进基因治疗研究转化为临床 干预为了支持题为“脂质疾病的AAV基因治疗”的P01，核心将生产和 质量控制研究和GMP工艺可比级AAV载体，用于临床前和IND 研究集中于治疗家族性卵磷脂-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）。核心还将转让AAV载体制造技术 在Penn开发的合同制造组织（CMO），以支持拟议的hoFH临床试验， 以及开发用于临床AAV载体产物的广泛表征的新测定法。

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.