Hutchinson-Gilford progeria syndrome (HGPS) is a rare progeroid syndrome caused by mutations in the LMNA gene. Currently there is no treatment available for HGPS but promising results from several studies using farnesyl transferase inhibitors (FTIs) on cells and animal models of HGPS have been published and a clinical trial using FTIs has been initiated in HGPS patients. However, published data from animal models treated with FTIs comes from studies where the treatment was initiated prior to significant disease development. In this study we utilized our inducible transgenic animal model of HGPS with abnormalities of the skin and teeth. Following phenotype development the transgenic expression was turned off and an improvement of the phenotype was noted already within four weeks of transgenic suppression. After 13 weeks the skin was almost indistinguishable from wild type skin. This study shows that in these tissues the expression of the progeria mutation does not cause irreversible damage and that the reversal of disease phenotype is possible, which gives promise for treatment for this disease.
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