Core A - Vector Core

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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.