Treatment+for+HGPS

Treatment for HGPS

Unfortunately there is no known cure for HGPS. In regards to treatment however, there are three therapies currently being investigated for their ability to inhibit post-translational farnesylation of progerin, the known disease causing protein of HGPS (8). These therapies, which are circled with their respective numbers on the farnesylation pathway, include:

1. Pravastatin (inhibits HMG-CoA reductase) 2. Zoledronate (a bisphosphonate that inhibits farnesyl pyrophosphate synthase) 3. Lonafarnib (farnesyltransferase enzyme inhibitor otherwise known as FTIs) With the inhibition of farnesylation, the amount of progerin accumulating in the nuclear membrane can decrease. Although it doesn't prevent the production of the mutant protein, it helps to reduce the dramatic abnormal morphology of the cell.

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(8) Brown, T., Collins, F., Gordon, L. (2003 updated 2011). //Hutchinson-Gilford Progeria Syndrome.// Retrieved from http://www.ncbi.nlm.nih.gov/books/ NBK1121/