Lessons from a rare disease

[摘要] (cont.) The story of its creation when Sam Berns, son of doctors Leslie Gordon and Scott Berns, was diagnosed with progeria in 1998, is also the story of the birth of modern progeria research in the U.S. Research highlighted in this thesis includes progeria;;s cardiovascular complications in transgenic mice; the discovery that progeria;;s symptoms can be reversed; clinical trials testing farnesyltransferase inhibitors or FTIs, statins and bisphosphonates, and all three together; the search for a cure; and the presence of progerin in the skin cells of healthy people.

[发布机构] Massachusetts Institute of Technology