Science's Best Friend

Last month in the Wall Street Journal, the newest advances in gene-editing technology were discussed. Amy Dockser Marcus reports that researchers at UT Southwestern Medical Center have utilized CRISPR/Cas9 to cure dogs suffering from muscular dystrophy. This is a genetic disease that causes deterioration of muscles by preventing the production of dystrophin. This experimental gene therapy successfully restored dystrophin production in various muscles of dogs, including the heart and diaphragm, with results as high as 90% restored function. The senior author of the research paper, Eric Olson, explains, “It’s like putting a good spare tire on a car. It’s not as good as the original, but it gets you where you want to go”.

Dogs Suffering from Muscular Dystrophy Find Relief Using CRISPR

            This is just one example of new gene therapies experimenting with the CRISPR/Cas9 system to cure genetic diseases. CRISPR, which stands for clustered regularly interspaced short palindromic repeats, is bacteria’s natural mechanism for developing resistance to viral infections. Cas9 is an associated endonuclease enzyme which cuts DNA at specific locations as directed by guide RNA. Upon discovery of this system, researchers were able to introduce this protein technology into eukaryotic cells. The repair system can also been adapted so that when a specific DNA sequence is determined to be compromised, such as the mutated dystrophin gene, guide RNA leads Cas9 to the affected area, which is then cut, and the cell’s repair machinery can then disrupt, delete, or even correct the mutated gene with a synthetic RNA template. This revolutionary system has offered hope for many genetic diseases, from muscular dystrophy to cystic fibrosis, as it attacks the mutations at the genetic level and would cure the underlying root cause of the diseases. 

How the CRISPR/Cas9 System Functions in DNA 

           

 Although this biochemistry discovery is shed in a positive light in the article as it helped improve the livelihoods of several dogs and holds promising potential for future cures in humans, there are still traces of chemophobia in the article. While discussing the CRISPR/Cas9 system the author asserts, “Its development…has also sparked concerns about unintentional cuts and deletions of DNA”. Despite there being no negative results or side effects in the dogs who received the treatment, the possibility that dangerous outcomes could occur is still discussed at some length. This is an example of chemophobia as the idea of chemical treatments often has a negative connotation even though most practices are safe and even life-saving, such as CRISPR. Strict regulations and standards are in place to avoid such possibilities. Just as UT first researched on cells, then mice, and then dogs, they still have many phases of research and regulations to complete before being allowed to complete clinical trials on humans. When these clinical trials do occur, they will be as safe and as harm free as possible, otherwise they would not be allowed to proceed. 

            The CRISPR/Cas9 system is a robust, chemical discovery that could save people’s lives.  It is being developed as a gene-therapy tool for a wide range of diseases, and the researchers at UT Southwestern have helped out several dogs suffering from muscular dystrophy, and hopefully soon will be able to do the same for humans. 

Sources:

https://www.wsj.com/articles/crispr-used-to-repair-gene-mutation-in-dogs-with-muscular-dystrophy-1535652001

http://www.crisprtx.com/crispr-platform/crispr-cas9-gene-editing.php

https://www.cambridge.org/core/journals/mrs-bulletin/news/crispr-implications-for-materials-science