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Duchenne muscular dystrophy: genome editing gives new hope for treatment
  1. Vassili Crispi,
  2. Antonios Matsakas
  1. Molecular Physiology Laboratory, Centre for Atherothrombotic and Metabolic Disease, Hull York Medical School, University of Hull, Hull, UK
  1. Correspondence to Dr Antonios Matsakas, Molecular Physiology Laboratory, Centre for Atherothrombotic & Metabolic Disease, Hull York Medical School, University of Hull, Hull, HU6 7RX, UK; Antonios.Matsakas{at}hyms.ac.uk

Abstract

Duchenne muscular dystrophy (DMD) is a progressive wasting disease of skeletal and cardiac muscles, representing one of the most common recessive fatal inherited genetic diseases with 1:3500–1:5000 in yearly incidence. It is caused by mutations in the DMD gene that encodes the membrane-associated dystrophin protein. Over the years, many have been the approaches to management of DMD, but despite all efforts, no effective treatment has yet been discovered. Hope for the development of potential therapeutics has followed the recent advances in genome editing and gene therapy. This review gives an overview to DMD and summarises current lines of evidence with regard to treatment and disease management alongside the appropriate considerations.

  • Duchenne Muscular dystrophy
  • gene therapy
  • genome editing
  • skeletal muscle

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Footnotes

  • Contributor VC and AM developed the idea and layout of this review. VC wrote the first draft of the manuscript. VC and AM revised and edited the final draft of the manuscript and approved the final text for submission.

  • Funding AM’s work has been supported by the European Union and The Royal Society (grants: FP7-PEOPLE-PCIG14-GA-2013-631440, 2014-2018 and RG140470 Research Grant, 2015-2016) and the Hull York Medical School. VC was funded by the INSPIRE project through Wellcome Trust at the Hull York Medical School.

  • Competing interests None declared.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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