An Update on Progress in Treating Progeria

We should be at least a little interested in the accelerated aging condition Progeria (or HGPS), for as this ScienceDaily article points out: "Recent research indicates that all people, not just children with HGPS, produce small amounts of [the truncated Lamin A protein called]progerin, and that this mutant protein may play roles in aging or longevity ... The discovery of the gene responsible for the disease five years ago led scientists to the experimental drug that is now being evaluated in 28 children ... The 2003 discovery of the HGPS gene, named lamin A (LMNA), laid the groundwork for the clinical trial. ... scientists found that the minute change in the LMNA gene's DNA sequence dramatically changed the way in which the sequence was spliced by the cell's protein-making machinery. The end result was the production of an abnormal lamin A protein that is missing a stretch of 50 amino acids near one of its ends. ... Years of basic research studies showed that the Lamin A protein production depends on the farnesyl group molecules' attaching themselves to the pre-lamin A protein. This attachment - and progerin production - can be blocked by a FTI drug."


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