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Four out of these fourteen are on the edge of being able to be edited, none the less it may take a while to test if it does work outside of the research and testing not conducted on a human embryo. These include amyloid transthyretin, beta-thalassemia, hemophilia type B, and Leber congenital amaurosis 10 (LCA10). With amyloid transthyretin, CRISPR-Cas9 is being tested to be used to do a gene knockout of the TTR gene. This gene knockout simply involves the deactivation or deletion of a sequence in a gene.
Next, beta-thalassemia, CRISPR-Cas9 is being used to change DNA sequences in the HBB gene to diminish the effects of the mutations. In this process, cells are removed, the HBB gene in the cell is edited, and then the changed cells are put back into the body. With hemophilia type B, ZFN editing (different from CRISPR-Cas9) is being used to replace the damaged F9 gene with a proper one in liver cells. It is important to note here that CRISPR-Cas9 will certainly not be the only gene editing technology around.
Others are already being tested, although CRISPR is the most advanced it is hard to see how much longer that will last. We will also see if the development of gene editing for creating “better” humans will be a pro or con in an ethical consideration list, possibly both. The new gene then creates specific coagulation factor proteins that allow the blood to clot. Lastly, with leber congenital amaurosis 10 (LCA10), CRISPR-Cas9 is supposed to remove the genetic mutation in the CEP290 gene.
An injection below the retina delivers the gene-editing therapy to the photoreceptor cells in the eye.
Even though the technology is new it is clear to see that gene editing has the possibilities to do great good by protecting people from harm without causing it. Something that I first thought of with when thinking about who this technology will help are the families that have a long line of diseases which have been consistently passed down from generation to generation. I think this also shows that there is a possibility to completely abolish some diseases all together and possibly create a fully disease-free world. In the future, gene editing may have the ability to fix other diseases that affect the world on a far larger scale, ones that affect millions of people every year. I believe this would include but certainly not be limited to Obesity, Diabetes, Cancer, Alzheimer’s and possibly even mental health. The limits with this technology are so impossible to even start to comprehend but it would be interesting to see how it could possibly play out with future generations as I research more into the ethical cons and of course both sides of the argument from the people themselves.
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