Core A - Vector Core

摘要 核心A：向量核心 宾夕法尼亚大学(宾夕法尼亚大学)载体核心是调查人员需要病毒为基础的关键资源 用于临床前和翻译研究的载体，用于开发获得性和获得性和非获得性基因治疗 遗传性疾病。核心的主要目标是提供源自 腺相关病毒(AAV)以促进基因治疗研究转化为临床 干预。为支持这篇题为“AAV基因治疗血脂紊乱”的P01，CORE将制作和 质量控制研究和GMP流程-临床前和IND支持的可比级AAV载体 家族性卵磷脂-胆固醇酰基转移酶(LCAT)缺乏症和纯合子的治疗研究 家族性高胆固醇血症(HoFH)。核心还将转让AAV矢量制造技术 在宾夕法尼亚大学开发为合同制造组织(CMO)，以支持拟议的HoFH临床试验， 以及为临床AAV载体产物的广泛表征开发新的分析方法。

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.