Comment
doi: 10.1016/j.stem.2016.03.004.
Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy
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- PMID: 27058929
- DOI: 10.1016/j.stem.2016.03.004
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Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy
Cell Stem Cell.
.
Free article
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.
Copyright © 2016 Elsevier Inc. All rights reserved.
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A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.Cell Stem Cell. 2016 Apr 7;18(4):533-40. doi: 10.1016/j.stem.2016.01.021. Epub 2016 Feb 11. Cell Stem Cell. 2016. PMID: 26877224 Free PMC article.
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