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Cell Stem Cell. 2016 Apr 7;18(4):423-4. doi: 10.1016/j.stem.2016.03.004.

Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy.

Author information

1
Department of Pathology, Institute for Stem Cell and Regenerative Medicine, Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA. Electronic address: ab67@uw.edu.
2
Department of Pathology, Institute for Stem Cell and Regenerative Medicine, Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA; Department of Bioengineering, Institute for Stem Cell and Regenerative Medicine, Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA; Department of Medicine/Cardiology, Institute for Stem Cell and Regenerative Medicine, Center for Cardiovascular Biology, University of Washington, Seattle, WA 98109, USA. Electronic address: murry@uw.edu.

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.

PMID:
27058929
DOI:
10.1016/j.stem.2016.03.004
[Indexed for MEDLINE]
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