Monday, April 17, 2017

Correcting Duchenne Muscular Dystrophy

Image result for CRISPR

The article "CRISPR Corrects Duchenne-Causing Mutations" discusses the research done using gene editing to treat a genetic disease. Duchenne Muscular Dystophy, DMD, is a X-linked disease that is fatal. Individuals with DMD have weakened skeletal systems and heart muscles which progressively get worse throughout their lives. Researchers have been studying a gene editing system for years called CRISPR-Cpf1. In a study at the University of Texas Southwestern Medical Center researchers found that this gene editing system reversed the effects of DMD. They took human stem cells and put them in a mouse model. After injecting the modified genes in the parents, the offspring of the genetically edited mice were noted to be stronger and had normal skeletal, muscle, heart and brain tissue. With this research, gene editing can be used to treat many fatal genetic diseases in the future.


  1. The research made on DMD has proven to be an important advancement in totally eradicating the disease. With further research and funding in gene editing, more diseases other than DMD will be able to be genetically modified to reverse the effects of the symptoms of other inherited diseases.

  2. This research can provide a lot of help in treating many disease and it's very interesting to know that this happening.

  3. Hopefully this advancement in gene editing makes waves when it comes to the involvement in reversing other diseases as well. The advancement with stem cells already shows the great changes in differentiation that are being made with the mice.