CRISPR helps heal mice with muscular dystrophy

31Jocelyn Kaiser, Science Magazine, December 31st, 2015. http://news.sciencemag.org/biology/2015/12/crispr-helps-heal-mice-muscular-dystrophy

Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy that primarily affects boys and usually results in death around age 25.  DMD is the result of defects or missing DNA in 79 exons that make up the dystrophin gene.  Due to the large size of the dystrophin genes, traditional gene therapy approaches have not been able to fully replace the faulty dystrophin gene.  In the December 2015 edition of Science, three independent groups report using CRISPR/Cas9 technology to remove defective exons of the dystrophin gene in young mice resulting in a truncated form of dystrophin (http://www.ncbi.nlm.nih.gov/pubmed/26721686, http://www.ncbi.nlm.nih.gov/pubmed/26721684, http://www.ncbi.nlm.nih.gov/pubmed/26721683).  While the treated mice showed considerable improvement compared to the controls, the treatment cannot be considered a cure since they did not perform as well on muscle tests as normal mice.  Despite its shortcomings, this technique 31could improve the quality of life for those living with DMD.

Author: Advanced Analytical

Advanced Analytical Technologies, Inc. (AATI) simplifies complex genomics workflows to accelerate research and discovery in pharmaceuticals, life science, biofuels, biotechnology and healthcare.

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