نتایج جستجو برای: fshd

تعداد نتایج: 347  

2013
Laetitia Davidovic Nelly Durand Olfa Khalfallah Ricardo Tabet Pascal Barbry Bernard Mari Sabrina Sacconi Hervé Moine Barbara Bardoni

The Fragile X-Related 1 gene (FXR1) is a paralog of the Fragile X Mental Retardation 1 gene (FMR1), whose absence causes the Fragile X syndrome, the most common form of inherited intellectual disability. FXR1P plays an important role in normal muscle development, and its absence causes muscular abnormalities in mice, frog, and zebrafish. Seven alternatively spliced FXR1 transcripts have been id...

2015
Eugénie Ansseau Jacqueline S. Domire Lindsay M. Wallace Jocelyn O. Eidahl Susan M. Guckes Carlee R. Giesige Nettie K. Pyne Alexandra Belayew Scott Q. Harper

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 or embedded within D4Z4 repeats. In the first published model, we used adeno-associated viral vect...

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