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Gene mutation causes progressive changes to cell structure in children with Progeria

[Boston, MA June 8, 2004] Researchers today announced that a mutation of the Lamin A gene gradually causes devastating effects on cellular structure and function in children with Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria). The study was published in this week's Proceedings of the National Academy of Sciences (PNAS). Progeria is a rare, fatal genetic condition characterized by an appearance of accelerated aging in children.

"Although it is a rare disease, Progeria has long been considered to be a model for studying the mechanisms responsible for normal aging" said lead author Robert D. Goldman, Ph.D., Stephen Walter Ranson Professor and Chair, Cell and Molecular Biology, Northwestern University, Feinberg School of Medicine. "This study highlights the importance of the Lamin A gene in the maintenance of cell structure and function."

In April 2003, a team of researchers assembled by The Progeria Research Foundation (PRF), and including the National Human Genome Research Institute (NHGRI), one of the 27 institutes and centers that make up the National Institutes of Health (NIH), announced the discovery of the gene that causes Progeria. That study, published in the April 16th 2003 issue of Nature, found that the disease is not inherited, but instead is caused by chance mutations to the LMNA gene (Lamin A). The Lamin A protein is the structural scaffolding that holds the nucleus together, and is involved in gene expression and DNA replication.

In the PNAS study, the result of a collaborative effort launched by researchers from Northwestern, The Progeria Research Foundation and NHGRI, microscopic and molecular techniques were used to examine the nuclear structure of cells from children with Progeria. As the Progeria cells aged, there was a gradual increase in defects in their nuclear structure and function, reflecting an abnormal accumulation of the defective Lamin A protein. Very similar changes were seen in normal human cells
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Contact: Brooke Saltzer
bsaltzer@spectrumscience.com
202-955-6222
Progeria Research Foundation
8-Jun-2004


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