What Is Gene Editing, and How Does It Work?

gene therapy

What Is Gene Editing, and How Does It Work?

Key Points

  • Gene editing is a revolutionary technology that allows scientists to make specific changes to the DNA of living organisms.
  • The advent of CRISPR-Cas9 in the early 2010s revolutionized gene editing by allowing scientists to edit genes with precision.
  • Important applications of gene editing include curing genetic diseases, treating cancer, developing new drugs, improving crop traits, performing organ transplants, and protecting the environment.
  • The future of gene editing raises ethical questions that need to be carefully addressed to ensure responsible and equitable use of this technology.

Gene editing is a revolutionary technology that has captured the attention of scientists and the public alike. There’s just one question, though. What exactly is gene editing? How does it work? Despite the decades of research and experimentation behind this remarkable feat of science, its explanation is quite simple. Let’s explore the basics of gene editing and its history. Then, let’s delve into the process involved. From there, we’ll examine some notable applications that hold promise for shaping the future of healthcare, food production, and beyond.

5 Must-Know Facts About Gene Editing

  • Gene editing is a powerful process that allows scientists to make precise and potentially life-altering changes to the DNA of living organisms. While research dates back to the 1970s, it didn’t truly take off until the 2010s.
  • The CRISPR-Cas9 system is a widely used and highly trusted tool in the scientific field of gene editing. First patented in 2012, the tool acts as a pair of molecular scissors that cuts DNA at specific locations.
  • Gene editing has numerous applications throughout science, medicine, and agriculture. Some of these applications include treating genetic diseases, improving the longevity of crops, and advancing new drug development.
  • In recent years, scientists have positioned gene editing as a potential cure for cancer. The ability to splice, remove, or amend damaged genes could revolutionize cancer treatments and transform therapies into more targeted and effective treatments.
  • Ethical considerations and regulations are crucial in the continued use of gene editing. Its very existence raises important questions about the implications of altering the human genome. Going forward, gene editing will need to prioritize responsible and equitable application of this technology at every step of the process.

The Background of Gene Editing

Gene editing is a groundbreaking new technology taking the world of science and medicine by storm. In short, gene editing allows scientists to make specific changes to the DNA of living organisms. Think of DNA like a code. This code carries precise instructions for any living thing’s makeup as well as how it functions. DNA determines everything from our eye color to our height to our susceptibility to certain diseases. The ability to edit this code could impact the future of humanity more than scientists and doctors can even fully comprehend.

The background of gene editing goes hand in hand with our understanding of genetics — or genes — as a whole. Genetics is the study of how an organism’s traits are passed down from one generation of the species to the next. Likewise, genes are an organism’s specific predispositions for certain traits passed from parent to child. Today, scientists understand that genes are the building blocks of heredity. Think of it like a set of characteristics and instructions passed along from an organism to its offspring.

While the discovery and study of DNA dates back as far back as the 1860s, the history of gene editing is much more recent. It all began with the revelation of restriction enzymes in the 1970s. Researchers found out that these proteins, known as restriction enzymes, could cut and splice DNA at specific spots. This blew the door open for the future of genetic engineering, also known as gene editing: the cutting, pasting, and manipulation of DNA. The discovery of restriction enzymes leads directly to the history of gene editing.

A close-up human genome.
Gene editing dates back to the origins of DNA research in the 1860s.


History of Gene Editing

Discovering restriction enzymes was undoubtedly a milestone in the history of gene editing. However, it wasn’t until the development of the polymerase chain reaction (PCR) technique that the history of gene editing could truly begin. Back in the 1980s, scientists developed a technique called polymerase chain reaction (PCR). This PCR technique made it much easier to work with DNA by essentially cutting and pasting specific sections of it. Needless to say, this completely changed the game of gene editing.

Scientists continued to experiment with PCR and DNA throughout the 1990s and into the 2000s. However, research and experimentation were extremely hindered by a limited understanding of gene editing at the time. All they could do was reproduce large quantities of DNA fragments and analyze them in-depth. It would take decades for scientists and researchers to crack the next step in the equation. Thankfully, that next step finally came in the early 2010s with the invention of CRISPR-Cas9.

This key discovery is a hybrid of two different components: CRISPR and Cas9. CRISPR is a bacteria’s natural defense system, helping it to fend off viral attacks. Cas9 is an enzyme that acts like a pair of scissors that can make precise cuts in a molecule’s DNA. By combining the two, scientists can edit genes by creating a guide that matches a specific sequence in a DNA strand. This guide — made of RNA — transports Cas9 enzymes to a specific target location, then cuts and pastes them into the DNA strand. Let’s dive deeper into this specific process below.

CRISPR Therapeutics logo on phone screen.
Gene editing would be nothing without the CRISPR-Cas9 tool.


The Process Behind Gene Editing

Gene editing was a long, complicated, and expensive process before the advent of CRISPR-Cas9. Here’s a general overview of how it works. First, scientists design a guide RNA (or gRNA) — a small RNA molecule that matches a specific DNA sequence that scientists wish to edit. The gRNA then guides the desired Cas9 enzyme to a specific location in a DNA strand. Think of it like molecular GPS navigation that takes the enzyme straight to its destination.

When Cas9 reaches the target site, it makes a precise cut in the strand of DNA, acting as a surgeon’s scalpel. This is what’s known as a double-strand break. The cut sets off an alarm to fix the DNA, triggering the natural repair mechanisms of the cell as a result. These natural repairs can occur in one of two ways: either by introducing organic changes to the DNA sequence or by incorporating a new piece of modified DNA from the lab. The specific repair method used will depend on the overall goal of the gene editing process.

Organic changes are referred to as non-homologous end joining (or NHEJ). This involves cutting out the problematic gene and allowing the DNA to fuse back together as if the target had never existed in the first place. The other method is known as homology-directed repair (or HDR). This requires a set of repair instructions disguised in the form of a small piece of DNA from the lab. Scientists cut out the problematic gene, then introduce their set of specific changes in its place. It might sound simple, but this simplicity comes as a result of decades of exhaustive research.

Close-up photo of a strand of molecules.
CRISPR-Cas9 approaches gene editing on a molecular level.


Important Applications of Gene Editing

After the advent of CRISPR-Cas9, scientists quickly realized the immense potential of this revolutionary technology. Before long, scientists started using the process to edit genes in various organisms ranging from bacteria and plants to animals and even humans. Let’s take a look at a handful of the most important applications in recent years. This will give us a clear picture of what the future might hold. If scientists have already accomplished all that’s listed below, then just imagine what they might achieve in the years to come.

Curing Genetic Diseases

By correcting underlying genetic defects, gene editing offers the potential to cure debilitating genetic diseases. Through this delicate scientific process, experts can target, repair, or outright replace faulty genes responsible for conditions such as cystic fibrosis, sickle cell anemia, and muscular dystrophy.

Treating Cancer Patients

Editing genes can also enhance cancer treatment by developing more effective and targeted therapies than chemotherapy or radiation. With further research, scientists should be able to modify immune cells to better recognize and attack cancer cells. This has the potential to create more personalized cancer treatments with improved precision and efficacy in the future.

Developing and Testing New Drugs

Gene editing has the potential to accelerate research in both biotechnology and drug discovery alike. Through the gene editing process, scientists can potentially modify organisms to produce more valuable compounds such as pharmaceuticals or biofuels.

Gene therapy being conducted on cancer cells.
With further research, gene therapy could be the long-awaited cure for cancer and other illnesses.

©CI Photos/Shutterstock.com

Gene editing also helps scientists to create more precise models of diseases, enabling a better understanding of their mechanisms and testing potential therapies. This is not only more efficient but also more sustainable than the drug development and testing methods used today.

Providing Food Security

It’s not just medicine that can benefit from gene editing. Gene editing can also potentially reshape the agricultural industry by improving weak crop traits. From enhancing nutritional content to increasing disease resistance to overcoming environmental challenges, agriculturalists can help crops better adapt to the changing demands of planet Earth (and maybe even solve world hunger).

Performing Organ Transplants

The list of patients in need of organ transplants could stretch miles long. Of course, this shortage of organs is limited by the fact that human bodies can only accept organs that match their specific blood and body types. However, gene editing holds the potential to address the shortage of organs by transforming other mammals’ organs to match the human body. For instance, scientists have seen serious promise with experiments on pig organs.

Protecting the Environment

The potential uses of gene editing stretch beyond food and medicine. As it turns out, gene editing has the potential to aid in conservation efforts by preserving endangered species and restoring ecosystems.

Scientists believe they can modify genes to increase a certain species’ resilience, combat invasive species that threaten a species’ livelihood, or mitigate the impact of climate change on endangered species.

A suburban landscape at sundown.
Gene editing could protect future crops from the effects of climate change.

©Jaaske M/Shutterstock.com

The Future of Gene Editing

While remarkable, the act of editing genes raises some very important ethical questions. These serious questions are sure to shape the future of gene editing as we know it. We know that gene editing has immense potential in medicine, agriculture, and scientific research alike. It can help treat genetic diseases, develop more effective therapies, improve the resilience of crops, and advance our understanding of genes and diseases on the whole. However, the question remains: “Just because you can, does it mean you should?”

Such serious considerations are necessary to ensure the responsible use of this technology going forward. It’s not hard to see why science is so controversial for some. It raises key discussions about the ethics of enhancing human traits and capabilities. Theoretically, scientists could use CRISPR-Cas9 to eliminate genetic disorders or greatly enhance traits like intelligence. However, the ethical ramifications and societal implications need to be carefully addressed before scientists head down such a road.

All in all, this discovery has immense potential to transform the future of life on Earth completely. This is true not only for the human race but for the natural world as a whole. Alas, this significance comes with an array of ethical, legal, and social concerns. These concerns must be carefully navigated by scientists if they wish to ensure responsible and equitable use of this groundbreaking technology. It’ll be many years — if not decades — before gene editing hits the mainstream. However, its promising applications are not to be overlooked.

What Is Gene Editing, and How Does It Work?

To review: gene editing allows scientists to make precise changes to the DNA of living organisms. Like writing code, gene editing lets scientists modify DNA to remove or amend specific traits or faults. Pending further research, it could have profound effects on the future of organisms as we know it. Through this process, scientists can potentially cure diseases, treat cancer, test drugs, fortify crops, transplant organs, combat climate change, and beyond. However, this process comes with its fair share of ethical questions that need answers sooner rather than later.

Frequently Asked Questions

What is gene editing?

Gene editing is a powerful tool that allows scientists to make precise changes to the DNA of living organisms. It involves the use of the CRISPR-Cas9 system to cut DNA at specific locations and introduce desired modifications.

How does gene editing work?

Gene editing works by using an enzyme, specifically Cas9, guided by a small RNA molecule to target and cut an organism’s DNA at a specific location. The cell’s natural repair mechanisms then come into play, either rejoining the DNA ends or incorporating new genetic material provided by scientists. This process allows for precise modifications to the DNA sequence.

What is gene editing used for?

Gene editing has a wide range of applications in today’s day and age. It holds promise for treating genetic diseases, developing more targeted therapies for cancer, improving crop traits for agriculture, advancing drug discovery, and enhancing our understanding of genetics and disease mechanisms.

What are the ethical ramifications of gene editing?

Gene editing raises important ethical considerations. One major concern is the potential for altering the human genome, which means changes made to DNA could be passed down to future generations. Ethical discussions also revolve around the responsible use of gene editing, ensuring equitable access, and the potential for unintended consequences.

Is gene editing safe?

Gene editing is a rapidly advancing field, and safety is a crucial consideration. While extensive research and testing are conducted to ensure safety, there is still much to learn about its safety if used on the masses. Rigorous oversight and regulations are in place to minimize risks and ensure responsible use. Continued research, monitoring, and open discussions contribute to the ongoing assessment of safety in gene editing applications going forward.

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