Core C: Animal Models Core The Animal Models Core will support Projects 1, 2 and 3 to allow for the in vivo evaluation of AAV-based gene therapy for lipid disorders that include familial hypercholesterolemia and lecithin cholesterol acyltransferase deficiency. The Core has extensive experience with the in vivo characterization of vectors in various murine models of lipid disorders. In the context of this P01 application, the Animal Models Core will provide full support at all levels including: study design, coordination of nonhuman primate acquisition, breeding for mice, management of in-life phase of animal studies including organization of the study, technical support for procedures and harvesting of samples. The Core will maintain and expand as needed four colonies that include the LDLR-/-APOBEC1-/-, the LDLR-/-APOBEC1-/-human ApoB, the LCAT knockout and the LCAT knockout/hAPOAI transgenic mice to support the needs of Projects 1 and 2. The Core staff are trained to conduct animal studies under GLP and work closely with the Quality Assurance Unit. More recently, the Core successfully completed IND-enabling studies to support AAV8-mediated gene therapy for familial hypercholesterolemia.
Core C: Animal Models Core The Animal Models Core has significant expertise in mouse colony expansion and maintenance that will allow Projects 1 and 2 to conduct the proposed studies. The Core staff are highly experienced in animal study design, animal acquisition, animal dosing and in-life study support in both mice and NHP. Furthermore, the Core is well equipped to conduct IND-enabling studies that will allow the clinical translation of candidate gene therapy AAV vectors for liver diseases that include LCAT deficiency.
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