New approaches to progeria

MW Kieran, L Gordon, M Kleinman - Pediatrics, 2007 - publications.aap.org
Progeria (Hutchinson-Gilford progeria syndrome) is a rare genetic disorder that offers
considerable insight into the biology of premature aging. This review summarizes the clinical
characteristics of this disease and the underlying mutation in the lamin A (LMNA) gene that
results in this phenotype. Modifications in the processing of prelamin A through alterations in
farnesylation are detailed, because this pathway offers a possible drug target. Finally,
discussion of an ongoing clinical trial for these children, including possible parameters for …