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Getting from 'Close' to 'Cigar' Via Alternate Splicing

Antisense Oligonucleotide Helps Severe Spinal Muscular Atrophy

By Anette Breindl

Monday, March 14, 2011
Scientists at Isis Pharmaceuticals Inc., with both industrial colleagues from Genzyme Corp. and academic colleagues from Cold Spring Harbor Laboratory and Harvard Medical School, have managed to improve the symptoms of spinal muscular atrophy (SMA) in a mouse model of the disorder by treating the animals with an antisense oligonucleotide. The work, Frank Bennett, Isis senior vice president of research, told BioWorld Today, "sets the stage for going forward into the clinic" with the compound, which the company hopes to do later in 2011.

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