Search Results
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Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD
Jan 17, 2018, 9:47 PM
A publication from Scott Harper's lab reporting on the use of an AAV vector to test the safety of two miRNAs in silencing DUX4.
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MRI change metrics of facioscapulohumeral muscular dystrophy: STIR and T1
Dec 16, 2017, 8:50 PM
Follow-on publication for previously funded grant.
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Research Publication: Catching DUX4 red (or green)-handed
Sep 2, 2015, 12:50 PM
Researchers at the University of Washington follow DUX4 - the real DUX4 - that's cool.
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Research Publication: A glimpse into SMCHD1 function
Aug 12, 2015, 12:35 PM
Researchers in Australia have carried out some important experiments towards our understanding of the FSHD2-causing gene, SMCHD1.
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Why is FSHD so variable?
Oct 30, 2014, 10:41 AM
Spoiler alert: it's because of epigenetics.
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Regeneration of Human Muscle in Mice
Jan 18, 2014, 7:36 PM
Dr. Kathryn Wagner provides a much needed tool.
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Science journal promotes transparency of data
Jan 17, 2014, 6:17 PM
Science joins Nature Publishing Group to Improve Reproducibility.
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High-throughput screening identifies inhibitors of DUX4-induced myoblast toxicity
Jan 7, 2014, 9:45 AM
We have developed a high-throughput screen based on the toxicity of DUX4 when overexpressed in C2C12 myoblasts, and identified inhibitors of DUX4-induced toxicity from within a diverse set of 44,000 small, drug-like molecules.
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New 4q35 Links to FSHD
Nov 11, 2013, 8:54 AM
Dysregulation of 4q35- and muscle-specific genes in fetuses with a short D4Z4 array linked to FSHD.
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Threats to Validity in the Design and Conduct of Preclinical Efficacy Studies
Jul 30, 2013, 6:30 PM
Unintentional bias and repeatability for preclinical studies has been gaining more and more attention as of late.
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WNT/β-catenin signaling suppresses DUX4 expression and prevents apoptosis of FSHD muscle cells
Jun 22, 2013, 5:32 PM
WNT/β-catenin signaling suppresses DUX4 expression and prevents apoptosis of FSHD muscle cells
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Longitudinal Features of Stir Bright Signal in FSHD
Jun 7, 2013, 5:25 PM
Magnetic resonance imaging shows short tau-inversion recovery (STIR) brightness in autosomal dominant FSHD1 suggestive of active inflammation/injury.
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Nature Journals Aim to Improve Reproducibility
Apr 18, 2013, 5:05 PM
Nature Journals Aim to Improve Reproducibility.