Abstract
Antisense oligonucleotides (ASOs) can be used to alter the splicing of a gene and either restore production of a required protein or eliminate a toxic product. In this issue of Genes & Development, Hua and colleagues (pp. 1634-1644) show that ASOs directed against an intron splice silencer (ISS) in the survival motor neuron 2 (SMN2) gene alter the amount of full-length SMN transcript in the nervous system, restoring SMN to levels that could correct spinal muscular atrophy (SMA).
Publication types
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Comment
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Research Support, N.I.H., Extramural
MeSH terms
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Alternative Splicing / drug effects
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Alternative Splicing / genetics
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Animals
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Disease Models, Animal
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Humans
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Introns
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Mice
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Muscular Atrophy, Spinal / genetics
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Muscular Atrophy, Spinal / metabolism*
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Muscular Atrophy, Spinal / therapy
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Nerve Tissue Proteins / genetics
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Nerve Tissue Proteins / metabolism
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Oligonucleotides, Antisense / chemistry
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Oligonucleotides, Antisense / genetics
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Oligonucleotides, Antisense / metabolism*
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Oligonucleotides, Antisense / pharmacology
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RNA Splicing / drug effects
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Survival of Motor Neuron 2 Protein / genetics
Substances
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Nerve Tissue Proteins
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Oligonucleotides, Antisense
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Survival of Motor Neuron 2 Protein