Until recently, the only method for modifying DNA was
the CRISPR-Cas9 system. However, the new Salk technology is much more efficient
than any other method because it allows the incorporation of DNA into non-dividing cells. The cells of the heart, brain, eye, and pancreas do not
divide again and are locked in G0. In order for this to be
accomplished a specific pathway called NHEJ was targeted by researchers. This
pathway repairs routine breaks in DNA sequences by rejoining original strand
ends. Researchers constructed a new complex called HITI, homology-independent
targeted integration, used to insert genes into the cells. The complex is made
up of different nucleic acids. Then, they used a virus to implant the HITI inside
the cell to transfer the genetic information to neurons that were derived from embryonic
stem cells from humans. Scientists then
completed a genetic modification in adult mice and it was successful. After
that, they tried to use the technique as a gene-replacement therapy method. The
researchers decided to work with mice that were born blind. They successfully
delivered the gene Mertk, one of the genes that, when damaged in humans, causes vision loss. The gene was inserted into the genome of the mice at just
three weeks old and by the time they were eight weeks old the mice showed
response to light. Furthermore, testing was continued and healing was observed in the retinal cells of the mice. I feel that many people will
benefit from this new gene-replacement therapy process. Individuals can be
assisted by a gene when having a disability, such as severe eye loss. Also, one that suffers from serious head trauma can gain some of his or her brain cells
back. Consequently, he or she will recover from a concussion quicker. This is only the beginning of the
great things science can do to help people suffering from genetic damage. Gene
therapy is going to revolutionize healthcare in the future.
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