Gene Editing in Living Organisms

555px-Benzopyrene_DNA_adduct_1JDGGenetic disease has long been regarded as incurable in the scientific community. Within today’s standards of care, diseases stemming from genetic mutations or  erroneous changes in the DNA sequence, have only been managed by dealing with and reducing symptoms. Researchers from the lab of Daniel Anderson at The Massachusetts Institute of Technology have successfully taken the next step in fighting genetic diseases.

The lab has employed a novel system of gene editing using a technique called CRISPR. This technique is much like the find and replace function found on a word processor. The scientists were able to selectively find the piece of mutated DNA in mice, cut it out, and replace it with the correct sequence of DNA. This treatment does not only deal with the symptoms, but addresses their root cause- the faulty programming of a mouse’s cells.

In their sample, the researchers were able to remove the faulty DNA resulting in a rare liver disease that will inevitably lead to the mice’s death. With successful use of the CRISPR technique, the mice effected by this mutation were cured of their disease.

The implications of this research are profound. Terrible diseases such as Cystic Fibrosis, Tay Sachs, and Huntington Chorea could potentially be cured using the same exact method. This could potentially save a large portion of the afflicted population from a painful and inevitable death at the hand of their genetic disease.

Furthermore, this technique could be employed controversially in the future. At this point, only disease states have been manipulated. However, what if this same technique were employed on healthy individuals to give them more favorable genetic traits? With successful development of this technique, humans could selectively modify their genes to alter the way they look, behave, or perform. Surely regulation will be required to determine what gene modification is ethical using the CRISPR technique.


Hao Yin, Wen Xue, Sidi Chen, Roman L Bogorad, Eric Benedetti, Markus Grompe, Victor Koteliansky, Phillip A Sharp, Tyler Jacks, Daniel G Anderson. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nature Biotechnology, 2014; DOI: 10.1038/nbt.2884

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