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Fig. 5 | Stem Cell Research & Therapy

Fig. 5

From: A non-viral genome editing platform for site-specific insertion of large transgenes

Fig. 5

Schematic representation of disease modelling for FSHD, and proposed methodology for potential drug screening. a The genetic defect in the DUX4 gene present in the repeats of the macro-satellite array (D4Z4) at chromosome 4q35 leads to array contraction to < 11 repeats and chromatin relaxation causing aberrant expression of the transcription factor DUX4 causing FSHD disease. b An illustration of transiently testing our reporter construct

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