On November 13th, in Oakland California, a man named Brian Madeux received a gene editor via IV. Mr. Madeux has Hunter syndrome, a degenerative disease that is caused because the patient lacks an enzyme that breaks down mucopolysaccharides. There is currently no cure for Hunter syndrome and due to damages to the lung, bones and heart most who have this disease have shortened life spans. This disease can be managed by injections that contain the enzyme they are missing but they still accumulate damages to their bones, heart, lungs and central nervous system. Mr. Madeux received a gene editor called zinc finger, this snips the DNA and inserts the gene he lacks. This gene editor was delivered using a non-pathogenic and non-proliferating virus. The company Sangamo, who is conducting this study, will monitor Mr. Madeux periodically to see if the gene editor inserted the correct gene.
While there are definite positives to gene editing, such as the potential to cure Hunter syndrome, there are potential ethical questions that are brought up by the prospect of editing our genes and these questions are becoming ever more relevant due to the fact that gene editing in living humans is no longer an idea but a reality. Ethical questions such, as where do we draw the line of what we are going to edit and not? Who is going to determine that line? How much will this cost? How much of our DNA should we control? Do we then edit our germ cells?
There are many ethical questions that need to be addressed when technology and biology advance. For me, in short, I think that the prospect of being able to help people with their debilitating and potentially life threatening diseases is a wonderful thing to strive for. With that being said, it's not as simple as just wanting to help people. A lot of if's and's or but's come into play and while gene editing was created to do good, there could be many situation where it can be used for bad or raise a lot of questions that no one is able to answer.
Bibliography
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I - Full Text View. (n.d.). Retrieved November 21, 2017, from https://clinicaltrials.gov/ct2/show/NCT02702115?term=sangamo&draw=1&rank=2
Genome Editing :: Sangamo Therapeutics, Inc. (SGMO). (n.d.). Retrieved November 21, 2017, from https://www.sangamo.com/technology/genome-editing
Scutti, S. (2017, November 16). Gene editing, in a patient's body, attempted for 1st time. Retrieved November 21, 2017, from http://www.cnn.com/2017/11/15/health/first-in-body-edit-dna/index.html
After reading the sources you listed, I was happy to see that many of the ethical issues you brought up were well thought out prior to the procedure. The virus delivering the gene editing tools had a specificity for liver cells which would prevent most of the random edits in other parts of the body. This also means that the germ cells should not be altered and edits will not be passed onto his children.
ReplyDeleteIt was also estimated that even if only 1% of the liver cells were successfully edited, it would be enough for Mr. Madeux to stop using the very expensive enzymes. This would be financially beneficial for Mr. Madeux and also prove this technique as effective for future patients.
This brings science closer to having to face the ethics of treatment vs enhancement, but I do not think it is a problem in this case.
This is a really interesting point that you bring up. I think that it is important to acknowledge that there are very really health benefits by editing the genes of a living person but it can also be a cause of concern because of the different ethical issues that it brings up. It makes you questions how far are we willing to take this new technology and should there be restrictions on it being used only for life threatening illnesses or should it also be allowed for illnesses that affect the patients quality of life?
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