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Updated: Aug 30, 2025
Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart
Published on: September 2, 2021
ELISpot Assay for Gene Therapy in Large Animal Studies
Renata Mazurek1, Kiyotake Ishikawa2
1Cardiovascular Research Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Abstract:
Formation of neutralizing antibodies and cellular immune response with repeat adeno-associated virus (AAV) gene therapy dosing are critical concerns in translational, large animal studies. The enzyme-linked immunospot/immunosorbent spot (ELISpot) assay introduced a way to track B- and/or T-cell response to therapy over time at a protein level. We describe the protocol for this assay looking at relative interferon (IFN)-γ secretion in pre- and post-AAV injections in a pig model.
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