CRISPR/Cas9 gene editing corrects Duchenne muscular dystrophy in mice

Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in DNA, allowing the body’s DNA repair mechanisms to replace it with a normal copy of the gene. The benefit of this over other gene therapy techniques is that it can permanently correct the “defect” in a gene rather than just transiently adding a “functional” one, said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern and Chairman of Molecular Biology.

Using CRISPR/Cas9, the Hamon Center team was able to correct the genetic defect in the mouse model of DMD and prevent the development of features of the disease in boys, which causes progressive muscle weakness and degeneration, often along with breathing and heart complications.

Potential of CRISPR Gene editing

George Church believes that CRISPR gene editing can be used to modify the human genome. Initially it will cure many human diseases. Later it will be used to enhance human health by altering genes for longevity.

George Church plans to use CRISPR gene therapy to incorporate what they learn from the supercentenarian studies, long lived animals (tortoises) and whatever they must create new using synthetic biology.

George Church is a giant in gene sequencing, synthetic biology and DNA science. In the October, 2012 Discover Magazine, George Church described some ideas he has for achieving physical immortality (indeterminant lifespans) via Synthetic biology.

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