What Does Gene Editing Mean for the Future?

There is a fine line between genetic engineering and eugenics. While eugenics purports to improve the genetic quality of the human population, genetic engineering is the modification of a genetic composition by artificial means, sometimes to treat inherited diseases or conditions.
This close relationship has brought the ethics of gene editing to the forefront. However, at the same time, gene editing is being called one of the most significant discoveries in modern-day biology.
One technique being used, called CRISPR Cas9, is an inexpensive and relatively precise editing method that plays a vital role in several fields. The main benefit of this latest gene-editing technology is its efficiency. Previous gene editing technologies could take months or even years to achieve less exact results than are currently possible with CRISPR Cas9.

The technology, MercatorNet notes, is “based on a natural process and replicates how bacteria fight viruses. By using guide RNA—the same type of molecule that bacteria use to find and fight a virus, but that can also easily be made in the lab from DNA in a few steps—scientists realized that they could target any spot in the genome of a plant or animal and make a deletion or paste something else in.”
A lot of the conversation relating to CRISPR Cas9 unsurprisingly revolves around the potential it has for editing the genes of human embryos. That’s a scary prospect to many, and is the main reason ethics is a central concern in genetic editing.
However, as Daniel Bauer, a hematologist at the Boston Children’s Hospital, has commented, “[f]or the humble molecular biologist, it’s really an extraordinarily powerful way to understand how the genome works.”

Gene editing targeting RNA

One of the more recent CRISPR Cas9 advancements is that it can be used to track and target the movement of RNA in living cells. That’s significant because while researchers have long been looking at human DNA, which indicates an individual’s predisposition to disease and determines diverse characteristics like hair and eye color, RNA is another vital piece of the puzzle.

“We are just beginning to see the implications of genome engineering using the CRISPR technology, but many diseases, including cancer and autism, are linked to problems with another fundamental biological molecule: RNA,” said researcher Gene Yeo. He’s part of a team at the University of California in San Diego that has identified how to use gene editing technology on RNA.
According to the release, Yeo and his team are exploring the ability of the RNA-targeted Cas9 to alter and measure other features of RNA processing beyond the current localization. “Future development of this approach could shed new light on dysfunctional RNA processes implicated in cancer and neurodegenerative disorders such as spinal muscular atrophy, as well as neurodevelopmental disorders such as fragile X syndrome — the most common inherited form of mental retardation,” the statement reads.
“One potential application of this technique is to track RNA transport in diseased neurons over time in order to identify the molecular features of these diseases and support the development of therapies,” said study author David Nelles. “Just as CRISPR-Cas9 is making genetic engineering accessible to any scientist with access to basic equipment, RNA-targeted Cas9 may support countless other efforts for studying the role of RNA processing in disease or for identifying drugs that reverse defects in RNA processing.”
While the end result remains to be seen, the theory is that future developments could enable researchers to measure other features of RNA processing or support therapeutic approaches to correct disease-causing RNA behaviors.

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Securities Disclosure: I, Vivien Diniz, hold no direct investment interest in any company mentioned in this article.