Fig. 1. Generation of the ΔEx50 mouse model.

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Fig. 1. Generation of the ΔEx50 mouse model. Generation of the ΔEx50 mouse model. (A) Strategy showing clustered regularly interspaced short palindromic repeats/CRISPR-associated 9 (CRISPR/Cas9)–mediated genome editing approach to generate ΔEx50 mice. (B) Reverse transcription polymerase chain reaction (RT-PCR) analysis of muscle RNA using primers in exons 48 and 55 to validate deletion of exon 50 (ΔEx50). Bands for wild-type (WT) and ΔEx50 mouse dystrophin are 1045 and 936 base pair (bp), respectively. (C) RT-PCR products from ΔEx50 mouse muscle were sequenced to validate exon 50 deletion and generation of an out-of-frame sequence. (D) Hematoxylin and eosin (H&E) staining and immunostaining of dystrophin in the cardiac and skeletal muscles from WT (upper) and ΔEx50 (lower) mice at 2 months of age. (E) Western blot analysis of dystrophin (DMD) and vinculin (VCL) expression in the tibialis anterior and heart muscle from 2-month-old ΔEx50 mice. VCL expression in the skeletal muscle displays two isoforms compared to the cardiac muscle. (F) Serum creatine kinase (CK), a marker of muscle damage and membrane leakage, in WT, ΔEx50, and mdx mice at 2 months of age. n = 5. Data are represented as means ± SEM. Red arrow indicates dystrophin protein. Scale bars, 50 μm. Leonela Amoasii et al., Sci Transl Med 2017;9:eaan8081 Copyright © 2017 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works