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Journal article

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

Abstract:

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 peptide-oligonucleotide, Pip6a-morpholino phosphorodiamidate oligomer (PMO), which dem...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Accepted Manuscript

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Publisher copy:
10.1073/pnas.1605731113

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Department:
Oxford, MSD, Physiology Anatomy and Genetics
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Department:
Oxford, MSD, Physiology Anatomy and Genetics
Shabanpoor, F More by this author
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Department:
Oxford, MSD, Physiology Anatomy and Genetics
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Funding agency for:
Bowerman, M
Publisher:
National Academy of Sciences Publisher's website
Journal:
Proceedings of the National Academy of Sciences of the United States of America Journal website
Volume:
113
Issue:
39
Pages:
10962–10967
Publication date:
2016
DOI:
EISSN:
1091-6490
ISSN:
0027-8424
Pubs id:
pubs:645882
URN:
uri:55a77ba7-c4f1-4a62-bb54-37ccffff7e9b
UUID:
uuid:55a77ba7-c4f1-4a62-bb54-37ccffff7e9b
Local pid:
pubs:645882

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