Genome Editing Goes Hi-Fi

A minor change in the genome of a human being can separate the healthy to the diseased. Scientists have studied mutations for a long time, but now, scientists at Gladstone Institutes led by Bruce Conklin, MD, have found a way to capture and edit the human genome one letter at a time—a way that can cure genetic diseases by fixing genetic code. In the past, studying human genetics and genetic changes had been inefficient, but now they have tools and methods that are more efficient and accurate.

First of all, what is genome editing? Genome editing, or genome editing with engineered nucleases (GEEN) is a way of manipulating the DNA using biotechnology in which DNA is put into, replaced, or removed from a genome using created nucleases. Nucleases are enzymes capable of cutting the bonds of nucleic acids. Currently, there are four groups of engineered nucleases in use: Zinc finger nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs), the CRISPR/CAS system, and engineered meganucleases. The nucleases make double-stranded breaks (DSBs) at specific locations in the genome, and repair the breaks using natural processes. However, because genome editing is still relatively new in modern biology, the engineered nucleases have difficulty in finding the part of the genome to modify. Sometimes, the DNA is unable to repair correctly after the process, and the nucleases themselves also cause problems. Lastly, only less than half of the patients that have gone through genome editing have received the needed results.

In the case of the researchers at Gladstone, they needed a way to identify a single mutated cell within hundreds of normal and healthy cells quickly. They designed a special fluorescent probe to differentiate the mutated sequence from normal sequences. From this, the scientists were able to sort through the sets of sequences and find mutant cells. The ability to detect is more than one hundred times greater through this method than through traditional methods. This method is then applied to induced pluripotent stem cells (iPS) cells, cells generated from skin cells of human patients with the same genetic makeup as the patient. The team used TALENs to edit the genetic code in the cells. One day, the researchers hope to repair and get rid of mutations in the human genome through their method.

Because this topic is related to technology, which I enjoy learning about, and it contains the material currently being covered in class about DNA and mutations, I was very excited to have come across this article. It was very interesting to learn about the editing of the human genetic code. I did not know the human genome could be edited; I thought it would be at least five to ten years before this was possible, so I also had fun endeavoring in learning about genome editing. I realize that the current methods can be dangerous, unsuccessful, and probably very expensive, but I also hope that the use of genome editing may become more widespread in the future once the techniques are improved so more people can be cured of genetic diseases.

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