Ivana De Domenico

Dr. Ivana De Domenico and Her Featured Writings

The Little Victories of CRISPR

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Biomedical research first coined the phrase, “war on cancer” in 1971. Many breakthroughs in research emerged in this decade as a result of the ongoing fight against this disease. And though many innovations were made in treatment, the cure has so far proved elusive. In the modern era, the introduction of CRISPR-Cas9, a tool to edit DNA, provides medical professionals with new frontiers in how cancer is treated. CRISPR opens up the potential to prevent cancer and place us one step closer to a cure. Even though many are concerned about the tool, we’ve already seen a lot of progress toward these goals.

Ambitious Goals

Researchers aim to either prevent cancer from happening in the first place or beating it all together, a lofty goal to be sure. While some have complained about the lack of progress in the last couple of decades, people shouldn’t overlook the value taken from incremental breakthroughs that bring the medical field on step closer to curing this heartbreaking illness. For example, some researchers hope to use CRISPR to reduce the debilitating effects of chemotherapy for patients.

Eradicating Cancer

In the big picture, researchers hope that CRISPR will eliminate diseases or cure them. In particular, monogenetic disorders can be easily targeted and eradicated through its use. Once researchers understand it better, the tool will grow in potential because the instruction manual could be rewritten for every living creature. Many of the researchers involved in CRISPR have kept a close eye on the ups and downs of this research because it displays incredible potential for many things. A steady flow of research has produced confusing twists and turns, but it could be an interesting step forward for medical science.

Concerns of Precision

Good research doesn’t ignore the potential for problems, however. For example, some scientists have expressed concern for the precision of CRISPR’s edits when they attempt to repair cells. If they go off-target, the edits could produce terrible changes to the DNA. Unfortunately, research still hasn’t come far enough to understand the potential side effects that could arise for patients. Even despite the promise, researchers must proceed with some level of caution to avoid a disaster. Though CRISPR is undeniably powerful, medical professionals have a duty to ensure that it is used and studied responsibly.

Chemotherapy has lifesaving potential, but unfortunately, it’s a terribly imprecise and unsophisticated tool. The toxins chemotherapy pumps into the body to destroy the cancer cells also kill the healthy cells as collateral damage. The people who have watched a loved one receive chemotherapy understand how it can impede the flow of daily life. Some of the nasty side effects include hair loss, nausea and anemia. Through CRISPR, scientists hope to ease these effects and make treatment a less painful and more dignified process for patients.

Working Out The Issues With CRISPR

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CRISPR is an enzyme developed by scientists in an effort to remove potentially harmful genetic traits and paste the remaining sequences together. During the 1980s, researchers discovered that bacteria protect themselves by cutting and pasting themselves. When bacteria identify viral RNA molecules within its DNA, the Cas enzyme grabs the anomaly and divides it in two to prevent reproduction. Scientists called the act clustered regularly interspaced short palindromic repeats or CRISPR for short. However, as researchers attempted to replicate the process in the laboratory, they uncovered problems.

The CRISPR discovery gave researchers hope that they had uncovered an innovative means of editing DNA. If scientists could use the technique, they dreamed that the abnormal DNA sequences that cause a number of disease processes might be effectively removed to prevent their development. However, as research and experimentation continued, CRISPR caused gaps in the cell’s genome. Scientists from the Wellcome Sanger Institute in Great Britain published the finding in an issue of “Nature Biotechnology” explaining that potential drawbacks of CRISPR have not been formally noted.

In a laboratory setting, researchers used the Cas-9 enzyme to remove a specific gene in the embryonic stem cells of a mouse. However, the process did not go as predicted. The group soon found that large sequences of DNA were missing. If the scientists were using traditional methods of assessing the DNA sequences, they may not have discovered the error. The billions of altered cells could have devastating effects that might include making them susceptible to cancer development.

The study showed that researchers need to assess larger strands of DNA to determine why CRISPR did not perform as hoped. Instead of cutting one strand of the gene, the team cut both strands of the double helix, which was one of the reasons for the error. They also need to find ways to prevent the cellular DNA from attempting to repair itself, which led to the problem.

Representatives of companies using CRISPR reported that their scientists had not encountered the problem with the techniques they used. As such, the study did not make an impact on their research. However, the published findings caused an immediate and substantial financial loss for several CRISPR companies.

The drawbacks did not dismay the Wellcome Sanger scientists. They explain that new discoveries are not without problems. The group plans to continue working on the project and making improvements so that one day the technology might be a successful medical tool.

Using CRISPR To Detect Diseases

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The genome editing tool CRIPSR was introduced to promote a new phase in genetic technology, but the tool is being adapted for another purpose. It may now be possible to use CRISPR to diagnose patients, giving doctors the ability to detect diseases and various medical conditions much sooner. This is exciting, because earlier detection means medical conditions can be treated before they worsen, which improves the chances of full recovery.
Jennifer Doudna, a researcher at the University of California at Berkeley, helped to found the company responsible for CRIPSR. She is also on the team behind this new application for the tool. In conjunction with Doudna, a University of California at Berkeley researcher, Mammoth Biosciences has announced plans to create easy to use diagnostic kits using Crispr technology. Once released, the kits could allow the early diagnosis of diseases in hospitals and even at home.
CRISPR is programmed to identify a specific piece of DNA and, when used in conjunction with certain proteins, it can actually repair genetic defects. Researchers hope this technology will advance to the point at which it will also be able to cure diseases in a similar way. When combined with the Cas12 or Cas13 proteins, CRISPR can already identify DNA left behind by viruses. In this way, it can possibly detect the Zika virus and some types of cancer.
Doudna isn’t alone. Researchers at Massachusetts Institute of Technology and Harvard University are each working on similar technologies. It will remain to be seen who perfects the application first, though papers have already been published to demonstrate that the technology does work. In Doudna’s paper, she found that the CRISPR-Cas12 system could spot the HPV virus. In her research, Doudna used a “reporter molecule” that exhibits a fluorescent signal once cut. She released the molecule into vials of human cells and found that only those cells infected with HPV glowed.
Meanwhile, the MIT group published a paper revealing that the CRISPR could be adapted to detect multiple diseases at once. Led by Feng Zhang, the MIT researchers also said that the kit could be manufactured for as little as $2 per test. This would go a long way toward helping people in low-income countries, where access to advanced diagnostic equipment is limited.
If CRISPR technology can be adapted in this way, it could change how we treat diseases. By treating illnesses sooner and more inexpensively, quality healthcare becomes more widely available.

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