title: Cardiovascular insights from a premature aging syndrome: a translational story.
abstract: Good morning. I am delighted to join you this morning. Today I'd like to tell you an interesting translational research story whereby a rare disease can be an example for how scientific advances can lead to acceleration of new therapies. This story will include some genetic sleuthing, a creative interdisciplinary team, a foundation of protein biochemistry, cutting-edge cell biology, creation of a mouse model, designer drug development, some good luck along the way, kids enrolling in a clinical trial from around the world, and finally, unexpected consequences for a condition that affects all of us.
authors.affiliation: Brigham and Women’s Hospital, 75 Francis St., Boston, MA 02115, USA.
authors.forename: Elizabeth G
authors.initials: EG
authors.lastname: Nabel
mesh_terms: D019588:Aging, Premature / Q000235:genetics / Q000503:physiopathology*; D000818:Animals; D002319:Cardiovascular System / Q000503:physiopathology*; D004195:Disease Models, Animal; D051231:Farnesyltranstransferase / Q000037:antagonists & inhibitors; D006801:Humans; D034904:Lamin Type A; D051379:Mice; D008822:Mice, Transgenic; D009154:Mutation / Q000235:genetics; D009687:Nuclear Proteins / Q000235:genetics; D010641:Phenotype; D011371:Progeria / Q000235:genetics / Q000503:physiopathology*; D011498:Protein Precursors / Q000235:genetics; D057170:Translational Research, Biomedical
chemical_list: D034904:Lamin Type A; D009687:Nuclear Proteins; D011498:Protein Precursors; C090764:prelamin A; D051231:Farnesyltranstransferase