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

On the way to Controlling Genomes

Genome editing is the control of adding, deleting, activating, or suppressing specific genes on DNA sequences.  Researchers have recently developed a new technique for genome editing.  The technique involves a system known as CRISPR-Cas (or clustered regularly interspaced short palindromic repeats-Cas).  CRISPR is a system used by bacteria to defend against viruses and other invaders; it targets and cuts DNA in a sequence-dependent manner to turn off or on genes that could harm the bacteria.

            Researchers are now utilizing this system to better understand and develop new ways to manipulate genes.  In addition to understanding and manipulating genes, the CRISPR-Cas system allows for increased accuracy and precision when targeting DNA.  This research has the potential to change the world.  Crops could be specifically altered to reduce or even grant immunity to diseases.  This could eventually be developed to work on people: genetically tailored drugs for your specific genome or changing you genome to grant immunity/resistance to disease.  One scientist, George M. Church, even predicted the possibility of de-extinction, human enhancement (develop human bodies suited for space and other hostile environments). Of course as exciting as gaining control over the human genome is, it raises the questions is it safe, effective, and morally right?  We are going to have to answer these question soon.

                

            I have always found control over the human genome to be fascinating.  The potential it has to improve the world is astronomical, which is why I thoroughly enjoyed reading this article.  It showed we are constantly advancing our techniques and approaches to handling the control of genes on DNA sequences.  When we finally gain the control over genes, we will be able to help the world and expand in all fields.  

Article: http://www.sciencedaily.com/releases/2014/10/141021141752.htm

Related Article:  http://www.thecrimson.com/article/2014/10/21/medical-professor-genome-editing/

Genetics breakthrough enables scientists to edit any part of human genome, report claims

According to The Independent a  new technique, known as Crispr has been discovered in Britain. This would allow scientists to engineer parts of the human genome with precision and accuracy. Originally developed to alter the DNA of crops and livestock, it is now being looked at to treat diseases such as cancer and inherited genetic disorders. Crispr works by using an RNA guide molecule that can be programmed to match any unique DNA sequence. The molecule is attached to a special enzyme that cut both strands of the DNA double helix. After this is done the then copied DNA is inserted back inot the double helix. Then the defecrtive DNA is deleted.  

Link: 
http://www.foxnews.com/science/2013/11/07/report-genetics-breakthrough-enables-scientists-to-edit-any-part-human-genome

Related Links:
http://www.independent.co.uk/news/science/exclusive-jawdropping-breakthrough-hailed-as-landmark-in-fight-against-hereditary-diseases-as-crispr-technique-heralds-genetic-revolution-8925295.html

http://www.genome-engineering.org/crispr/