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High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines

Abstract:

Myotonic dystrophy (DM) is a multi-system neuromuscular disorder for which there is no treatment. We have developed a medium throughput phenotypic assay, based on the identification of nuclear foci in DM patient cell lines using in situ hybridization and high-content imaging to screen for potentially useful therapeutic compounds. A series of further assays based on molecular features of DM have also been employed. Two compounds that reduce and/or remove nuclear foci have been identified, Ro 3...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1093/hmg/ddt542

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Department:
Oxford, MSD, Clinical Neurosciences
Robinson, TE More by this author
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Publisher:
Oxford University Press Publisher's website
Journal:
Human Molecular Genetics Journal website
Volume:
23
Issue:
6
Pages:
1551–1562
Publication date:
2013-10-30
Acceptance date:
2013-10-24
DOI:
ISSN:
0964-6906 and 1460-2083
Pubs id:
pubs:646420
URN:
uri:00137453-45e3-4c7d-a7d8-685b528ed197
UUID:
uuid:00137453-45e3-4c7d-a7d8-685b528ed197
Local pid:
pubs:646420
Keywords:

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