Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
SM Hammond, G Hazell… - Proceedings of the …, 2016 - National Acad Sciences
The development of antisense oligonucleotide therapy is an important advance in the
identification of corrective therapy for neuromuscular diseases, such as spinal muscular
atrophy (SMA). Because of difficulties of delivering single-stranded oligonucleotides to the …
identification of corrective therapy for neuromuscular diseases, such as spinal muscular
atrophy (SMA). Because of difficulties of delivering single-stranded oligonucleotides to the …
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