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Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
- Chalermchai Mitrpant,
- Paul Porensky,
- Haiyan Zhou,
- Loren Price,
- Francesco Muntoni,
- Sue Fletcher,
- Steve D. Wilton,
- Arthur H. M. Burghes
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- Published: April 22, 2013
- https://doi.org/10.1371/journal.pone.0062114