1. Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy 
Astrid Breitbart, Charles E. Murry 
Cell Stem Cell 
Volume 18, Issue 4, Pages (April 2016)
DOI: /j.stem
Copyright © 2016 Elsevier Inc. Terms and Conditions

2. Figure 1
Possible Therapeutic Approaches for Treating DMD with Gene Editing Using CRISPR/Cas9
Upper panel: deletion of exons 45–55 of the DMD gene through the introduction of two double-strand breaks with the Cas9 nuclease and two gRNAs flanking exon 45 and exon 55 and NHEJ DNA repair.
Bottom panel: possible approaches for the treatment of DMD patients with CRISPR/Cas9. Left: in vivo genome editing through systemic application of the Cas9 nuclease with two gRNAs via AAV vectors. Right: in vitro genome editing of patient-derived iPSCs and subsequent differentiation into skeletal muscle cells or cardiomyocytes for local or systemic cell therapy.
Cell Stem Cell  , DOI: ( /j.stem )
Copyright © 2016 Elsevier Inc. Terms and Conditions