Central Nervous System Disease
Data on central nervous system disease described by researchers at Cold Spring Harbor Laboratory
Published in Gene Therapy Weekly, September 2nd, 2010
New investigation results, 'Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model,' are detailed in a study published in Genes & Development. "Increasing survival of motor neuron 2, centromeric (SMN2) exon 7 inclusion to express more full-length SMN protein in motor neurons is a promising approach to treat spinal muscular atrophy (SMA), a genetic neurodegenerative disease. Previously, we identified a potent 2'-O-(2-methoxyethyl) (MOE) phosphorothioate-modified antisense oligonucleotide (ASO) that blocks an SMN2 intronic splicing silencer element and efficiently promotes exon 7 inclusion in transgenic mouse peripheral tissues after systemic...
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Source: Gene Therapy Weekly (2010-09-02)
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