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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

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Publisher copy:
10.1073/pnas.1605731113

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
More from this funder
Funding agency for:
Bowerman, M
Grant:
Career Development Fellow
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:
2016-01-01
Acceptance date:
2016-07-29
DOI:
EISSN:
1091-6490
ISSN:
0027-8424
Pmid:
27621445
Source identifiers:
645882
Language:
English
Keywords:
Pubs id:
pubs:645882
UUID:
uuid:55a77ba7-c4f1-4a62-bb54-37ccffff7e9b
Local pid:
pubs:645882
Deposit date:
2016-09-27

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