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Aberrant Splicing in Transgenes Containing Introns, Exons, and V5 Epitopes: Lessons from Developing an FSHD Mouse Model Expressing a D4Z4 Repeat with Flanking Genomic Sequences
The DUX4 gene, encoded within D4Z4 repeats on human chromosome 4q35, has recently emerged as a key factor in the pathogenic mechanisms underlying Facioscapulohumeral muscular dystrophy (FSHD). This recognition prompted development of animal models expressing the DUX4 open reading frame (ORF) alone o...
Autores principales: | Ansseau, Eugénie, Domire, Jacqueline S., Wallace, Lindsay M., Eidahl, Jocelyn O., Guckes, Susan M., Giesige, Carlee R., Pyne, Nettie K., Belayew, Alexandra, Harper, Scott Q. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4351184/ https://www.ncbi.nlm.nih.gov/pubmed/25742305 http://dx.doi.org/10.1371/journal.pone.0118813 |
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