ISSLS prize in basic science 2021: a novel inducible system to regulate transgene expression of TIMP1

摘要

Purpose Inflammatory and oxidative stress upregulates matrix metalloproteinase (MMP) activity, leading to intervertebral disc degeneration (IDD). Gene therapy using human tissue inhibitor of metalloproteinase 1 (hTIMP1) has effectively treated IDD in animal models. However, persistent unregulated transgene expression may have negative side effects. We developed a recombinant adeno-associated viral (AAV) gene vector, AAV-NF kappa B-hTIMP1, that only expresses the hTIMP1 transgene under conditions of stress.