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CSHL researchers demonstrate efficacy of antisense therapy for spinal muscular atrophy
Date:7/12/2010

Cold Spring Harbor, N.Y. The devastating, currently incurable motor-neuron disease spinal muscular atrophy (SMA) might soon be treated with tiny, chemically modified pieces of RNA called antisense oligonucleotides (ASOs).

Scientists at Cold Spring Harbor Laboratory (CSHL) and California-based Isis Pharmaceuticals have succeeded in reversing symptoms of Type III SMA, a relatively mild form of the disease, in mice by introducing an ASO into their spinal cords. The ASO fixes the molecular mistake underlying SMA by redirecting a cellular editing process called alternative splicing.

"Validating ASO efficacy in animal models is a crucial pre-clinical step before this strategy can be applied in SMA patients," says CSHL Professor Adrian Krainer, Ph.D. "We have now successfully demonstrated this therapeutic efficacy in the mouse nervous system. Although the mice only have the mild symptoms of Type III SMA, our treatment can effectively correct them."

Based in part on the team's findings, which appear online ahead of print on July 12th in Genes and Development, Isis selected an antisense drug candidate to move forward in development to treat SMA.

"SMA is the leading genetic cause of infant mortality and has limited treatment options for patients. With Dr. Krainer's lab at Cold Spring Harbor Laboratory, we have made significant progress in identifying a drug development candidate and conducting early preclinical studies to access its therapeutic potential," said Frank Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals. "We are committed to advancing this program toward the clinic."

SMA is caused by insufficient levels of a protein called Survival of Motor Neuron (SMN) in the spinal cord's motor neurons, which waste away along with the muscles that they can no longer control. The SMN1 gene, which produces the SMN protein, is missing or mutated beyond repair in SMA patients. Humans have a seco
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Contact: Hema Bashyam
bashyam@cshl.edu
516-367-6822
Cold Spring Harbor Laboratory
Source:Eurekalert

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