Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies

UMMS Affiliation

Department of Cell and Developmental Biology



Document Type



Bioinformatics | Cell Biology | Computational Biology | Developmental Biology | Genomics | Molecular Biology | Molecular Genetics | Musculoskeletal Diseases | Nervous System Diseases


Versatility of CRISPR/Cas9-based platforms makes them promising tools for the correction of diverse genetic/epigenetic disorders. Here we contrast the use of these genome editing tools in two myopathies with very different molecular origins: Duchenne muscular dystrophy, a monogenetic disease, and facioscapulohumeral muscular dystrophy, an epigenetic disorder with unique therapeutic challenges.

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Citation: Himeda CL, Jones TI, Jones PL. Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies. Trends Pharmacol Sci. 2016 Feb 22. pii:S0165-6147(16)00025-0. doi: 10.1016/j.tips.2016.02.001. [Epub ahead of print] PubMed PMID: 26917062. Link to article on publisher's site

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