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Article

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.

Proceedings of the National Academy of Sciences of the United States of America - 27 Sept 2016

Hammond Suzan M, Hazell Gareth, Shabanpoor Fazel, Saleh Amer F, Bowerman Melissa, Sleigh James N, Meijboom Katharina E, Zhou Haiyan, Muntoni Francesco, Talbot Kevin, Gait Michael J, Wood Matthew J A

Abstract excerpt

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 CNS, current approaches have been restricted to using invasive intrathecal single-stranded oligonucleotide delivery. Here, we report an advanced...

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Share to Muscular Atrophy, Spinal

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Survival of Motor Neuron 2 Protein

Source

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Identifiers

PMID: 27621445
DOI: 10.1073/pnas.1605731113
PMCID: PMC5047168

Genes

SMN1
SMN2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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