Engineered FSHD mutations results in D4Z4 heterochromatin disruption and feedforward DUX4 network activation
Author:
Funder
NIH
Japan Agency for Medical Research and Development
Publisher
Elsevier BV
Reference84 articles.
1. Population-based incidence and prevalence of facioscapulohumeral dystrophy;Deenen;Neurol.,2014
2. Current therapeutic approaches in FSHD;Wang;J. Neuromuscul. Dis.,2021
3. The D4Z4 repeat-mediated pathogenesis of facioscapulohumeral muscular dystrophy;van der Maarel;Am. J. Hum. Genet.,2005
4. Facioscapulohumeral muscular dystrophy and DUX4: breaking the silence;van der Maarel;Trends Mol. Med.,2011
5. Facioscapulohumeral muscular dystrophy family studies of DUX4 expression: evidence for disease modifiers and a quantitative model of pathogenesis;Jones;Hum. Mol. Genet.,2012
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1. Single-cell spatial transcriptomics reveals a dystrophic trajectory following a developmental bifurcation of myoblast cell fates in facioscapulohumeral muscular dystrophy;Genome Research;2024-05
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