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

Astrid Breitbart; Charles E Murry

doi:10.1016/j.stem.2016.03.004

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

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

Authors

Astrid Breitbart¹, Charles E Murry²

Affiliations

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.

PMID: 27058929
DOI: 10.1016/j.stem.2016.03.004

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.

Publication types

Comment

MeSH terms

CRISPR-Cas Systems*
Dystrophin / metabolism*
Exons
Genetic Therapy
Humans
Muscular Dystrophy, Duchenne

Substances

Dystrophin