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CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
Published on: September 14, 2019
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Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy
Astrid Breitbart1, Charles E Murry2
1Department of Pathology, Institute for Stem Cell and Regenerative Medicine, Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA.
Cell Stem Cell
|April 9, 2016
Abstract:
There is still no curative treatment for Duchenne muscular dystrophy (DMD). In this issue of Cell Stem Cell, Young et al. (2016) demonstrate a genome editing approach applicable to 60% of DMD patients with CRISPR/Cas9 using one pair of guide RNAs.
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