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Health News
Spinal muscular atrophy therapy possible
COLD SPRING HARBOR, N.Y., Nov 5, 2009 (UPI via COMTEX) -- U.S. medical
scientists say a molecule related to the common antibiotic tetracycline might be
useful in treating spinal muscular atrophy.
A research collaboration involving scientists from the Cold Spring Harbor
Laboratory, Paratek Pharmaceuticals and the Rosalind Franklin University of
Medicine and Science said the disease, which is currently incurable, is the
leading genetic cause of death in infants.
Spinal muscular atrophy is caused by mutations in a gene called SMN1, resulting
in a decrease in the levels of a protein in the motor neurons of the spinal cord
-- the cells that control muscle activity. Without the protein, the researchers
said, neurons degenerate and infants born with the mutations progressively lose
their ability to move, swallow and breathe.
The scientists said their new molecule boosts the levels of the SMN1 protein in
cells by fixing a mistake in a cellular processing mechanism called RNA
splicing. The researchers said they successfully created such a fix in both
mouse models of spinal muscular atrophy, as well as in cells isolated from
patients.
The study is reported in the journal Science Translational Medicine.
URL: www.upi.com
Copyright: Copyright 2009 by United Press International
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