Full-length dystrophin restoration via targeted exon addition in dmd-patient specific ipscs and cardiomyocytes

HIGHLIGHTS

• who: Rou Xiao and colleagues from the Center for Medical Genetics, School of Life Sciences, Central South University, Changsha, China have published the paper: Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes, in the Journal: (JOURNAL)
• what: In this study, an in situ genetic correction approach to restore the full-length dystrophin protein coding region in DMD-iPSCs was developed via targeted addition of the missing exon using CRISPR/Cas9. All in all, the study demonstrated that full-length dystrophin was restored in iPSCs and iCMs via targeted . . .
  

   

  

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