Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
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Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
Hammond SM, Hazell G, Shabanpoor F, Saleh AF, Bowerman M, Sleigh JN, Meijboom KE, Zhou H, Muntoni F, Talbot K, Gait MJ, Wood MJ. Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy. Proc Natl Acad Sci U S A. 2016 Sep 12. pii: 201605731. [Epub ahead of print]
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