Drug prevents cell abnormality leading to progeria
Aug 30, 2005 - 7:38:38 PM
BACKGROUND: One in 4 million children are born with progeria, a genetic disease marked by accelerated aging and early cardiovascular disease. The children suffer from dwarfism, baldness, wrinkles, hardened arteries and osteoporosis. Most die from heart disease before age 15.
The rare disorder stems from a mutation in a gene that produces an abnormal cellular protein, which attaches itself to structures in the cell's nucleus. The accumulated protein deforms the nucleus, sparking miscommunications with other cells and leading to the genetic disease.
FINDINGS: UCLA scientists studied cells isolated from people with progeria and cultured the cells with a drug that blocked the mutant protein from attaching to the cells' nuclei. The drug significantly reduced the number of human cells with misshapen nuclei. The UCLA team earlier used the same approach to improve the shape of abnormal nuclei from mice genetically engineered to develop progeria.
IMPACT: The findings offer new clues into how progeria develops and could lead to new drugs to treat the disease and its related disorders, including osteoporosis and hardening of the arteries. UCLA's next step will be to test whether returning the shape of the nuclei to normal stops development of progeria in mice.
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