Thursday, November 13, 2014

A New Effective Method of Genome-Editing

Research and technology in genetic modification and genome-editing is a contemporary field that is broadening its horizons at an exponential rate. Genome-editing often relies on the technique known as CRISPR/Cas9. This processes relies on two key tools of gene insertion. The first of which being an endonuclease enzyme to cut the DNA at specific points designated by the researcher. The second tool for CRISPR/Cas9 is the use of a promoter (a promoter is an on switch to turn on a designated gene). This process has been used by researchers to insert genes and to disable genes at the researcher's control. However, using this method in humans is risky. The use of a Cas9 to cut DNA at specific locations could also cut DNA at unintended locations, and the use of a promoter could also effect unintended genes.

A new method was tested and developed by the Stanford University Medical Center led by Mark Kay and Adi Barzel. The goal of their testing was to not only eliminate the use of nucleases and promoters for gene insertion, but to also cure hemophilia in mice. To do this they used a modified virus that was removed of all viral DNA and left only therapeutic DNA containing a blood clotting factor gene. This was inserted into mice and targeted the albumin gene. They then relied on genetic recombination of chromosomes to copy the genes inserted into the mice. They were able to cure hemophilia in both newborn and adult mice with the inserted blood clotting factor gene. This is revolutionary in that not only were they successful in curing a disease that effects human's worldwide, but also it eliminates the risk of the old method of gene insertion. Much more work, research, and testing must be done on the new method, but the future of gene therapy in humans is looking promising.

Article Link: http://www.sciencedaily.com/releases/2014/10/141029145444.htm
Supporting Link:http://www.nature.com/nature/journal/vaop/ncurrent/full/nature13864.html

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