Hutchinson Gilford Progeria Syndrome Research Paper

Abstract Hutchinson–Gilford progeria syndrome is an extreme rare genetic disorder that catastrophically influences children in pre-mature aging and dying in their teenagers. So far, all drugs used to treat this disease are focusing on reducing complications and extending the life spans of patients, but no treatment has been proven to treat this disease directly and effectively. Fortunately, the new drug named farnesyltransferase inhibitors appears recently in the research field of Hutchinson–Gilford progeria. My hypothesis is that farnesyltransferase inhibitors are effective and safe to treat children born with progeria. My first objective is to test the effectiveness of farnesyltransferase inhibitors in both animal and human models.
On the one hand, figuring out causes, mechanisms, and possible treatments, specifically farnesyltransferase inhibitors, will be a huge success and even a milestone in the clinical medicine. It will benefit children born with this genetic disorder. On the other hand, besides studying the disorder itself, scientists can find out some clues and hints about the normal aging process. It will be advantages for the whole human being to know their aging process. (Deepu 2015) So far, there is no treatment proven effective and curable for this disorder. The most common treatments aim at elongating children’s lives and reducing complications, such as cardiovascular diseases. Some medicines are suggested to prevent heart attacks and some high-nutrition supplements are recommended to prevent weight loss. Recently, scientists find a new drug called farnesyltransferase inhibitor that might be used to treat HGPS in the future. However, the effectiveness and safety of farnesyltransferase inhibitor are not well studied so far. Based on these unsolved problems, the objective of this mini grant proposal is to try to access the effectiveness and safety of farnesyltransferase inhibitors in both animal models and human cells. (Deepu