Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies

Charis L Himeda; Takako I Jones; Peter L Jones

doi:10.1016/j.tips.2016.02.001

Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies

Trends Pharmacol Sci. 2016 Apr;37(4):249-251. doi: 10.1016/j.tips.2016.02.001. Epub 2016 Feb 22.

Authors

Charis L Himeda¹, Takako I Jones¹, Peter L Jones²

Affiliations

¹ The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
² The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA. Electronic address: Peter.Jones@umassmed.edu.

Abstract

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.

Keywords: CRISPR; Cas9; DMD; FSHD; genome editing; muscular dystrophy.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Animals
CRISPR-Cas Systems*
Clustered Regularly Interspaced Short Palindromic Repeats
Homeodomain Proteins / genetics
Humans
Muscular Dystrophy, Duchenne / genetics*

Substances

Homeodomain Proteins

Abstract

Publication types

MeSH terms

Substances

Grants and funding