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Genetic technology is reshaping our understanding of the world. As you read this, scientists are uncovering fascinating ways to modify DNA. A prime example is CRISPR, an advanced gene-editing tool that has opened up new frontiers in genetic research. So revolutionary, in fact, that its inventors were awarded the Nobel Prize in Chemistry in 2020. This technology enables precise DNA modifications and is already contributing to the development of new cancer therapies. Experts believe it could eventually provide cures for genetic disorders.
Naturally, gene editing has sparked debates due to ethical concerns. Critics argue that scientists are 'playing god' with the genetic code. However, this technology has also fueled remarkable scientific breakthroughs. Below are ten of its most astonishing applications.
10. Genetically Modified Goats That Produce Cancer Drugs in Their Milk
In New Zealand, scientists have engineered goats to produce cancer-fighting drugs in their milk. These modified goats are specifically designed to generate cetuximab, a medication used to treat cancers of the colon and lungs. While the drug can cost up to $13,000 per month without insurance, the hope is that this new production method will significantly reduce the price, making the medication more affordable.
Producing cetuximab is a costly process due to its complex chemical composition, which requires the use of proteins derived from mouse cells for its production. However, these genetically modified goats present a new opportunity for the pharmaceutical industry to mass-produce cetuximab.
'It’s far more cost-effective to produce cetuximab in animals because their mammary glands can generate large quantities of proteins,' said Götz Laible, the lead researcher on the project at New Zealand’s AgResearch institute.
9. Scientists Are Storing Data Inside Living DNA
Data storage is a complex task. We rely on devices like hard drives, optical disks, and memory sticks to store vast amounts of data daily. But now, scientists are exploring alternative materials that might be more suitable for storage. Researchers in New York have developed a new approach using gene editing to store information in the DNA of living bacteria.
In 2021, a team at Columbia University showed that live E. coli cells could store up to 72 bits of data. A data file consists of a series of ones and zeros, and by inserting specific genes into the E. coli DNA, they were able to encode this binary data. They even managed to encode the phrase 'Hello world!' into the DNA of an E. coli cell, which they later decoded by sequencing the DNA.
DNA proves to be remarkably efficient for data storage. Biological proteins are capable of storing vast amounts of information. Scientists believe that if a strand of DNA were the size of a grain of salt, it could hold the equivalent of ten full-length films. Moreover, the technology to read and write DNA is advancing rapidly. However, DNA data storage is still in its early stages, so it's unlikely to become mainstream anytime soon.
8. Extending the Life of Terminally Ill Mice
Researchers at Harvard University have managed to extend the lifespan of dying mice through gene editing, more than doubling their life expectancy.
Under the leadership of Professor David Liu, the mice were deliberately given progeria, a rare genetic condition that accelerates aging in children. On average, children with progeria only live to about fourteen years old. The condition, caused by a rare genetic mutation, cannot be treated by traditional gene therapy. Instead, the Harvard team is working on altering the fundamental DNA code in those suffering from progeria.
This method was tested on the critically ill mice, and it notably increased their life spans. Initially expected to live only 215 days, the mice lived a median of 510 days. Liu and his team hope these results will lead to effective treatments for progeria and other similar genetic disorders.
7. Gene Therapy in One Eye Improves Vision in Both
Researchers have uncovered a gene therapy treatment for vision loss that, when administered to one eye, enhances sight in both. The therapy works by transferring genes from the treated eye to the untreated one, though eye specialists are still unsure of the long-term implications of this phenomenon.
The scientists were investigating a condition called Leber’s hereditary optic neuropathy (LHON), a rare and progressive vision loss disorder primarily affecting young men. LHON is caused by a genetic mutation that attacks and damages the retina's cells in the eye.
During a recent trial, 37 patients suffering from LHON received gene therapy injections in one eye. Surprisingly, after two years, 29 of the patients reported improved vision in both eyes. The scientists initially were puzzled by these results until they realized that the therapy's viral vectors were moving from one eye to the other.
When the same experiment was repeated on macaque monkeys, researchers found that the genes traveled down the optic nerve of one eye, crossed over to the other optic nerve, and then entered the second eye.
6. A Harmless Bull Without Horns
Researchers have discovered a method for creating bulls without horns by editing the father’s DNA. This new approach offers farmers a pain-free alternative to traditional dehorning practices. Currently, cattle must undergo a painful procedure to have their horns removed. This lengthy and difficult process is essential, however, as hornless bulls are not only safer for other animals but also easier to transport and take up less space at the feeding trough.
In 2016, two calves were born with a genetic mutation that prevents them from growing horns. This was made possible by introducing a small DNA sequence into the father’s cells. After testing the DNA of all three bulls, scientists confirmed that the genetic modification had been passed down to the calves without any unintended side effects.
'We’ve shown that it is possible to produce healthy hornless calves with only the intended genetic modification, and we’ve provided data to guide the evaluation process for genome-edited animals,' explained Alison Van Eenennaam, a specialist in animal science at the University of California, Davis.
5. Cows Are Genetically Engineered to Withstand Heat Stress
As temperatures rise, cows begin to suffer from the heat. Bovines are especially vulnerable to heat stress. When exposed to prolonged sunlight, cows lose their appetite, produce less milk, and struggle to conceive. For farmers, these effects can be devastating. Heat stress costs the U.S. dairy industry an estimated $900 million annually, and in poorer nations, where farmers may only have a few animals, it can lead to financial ruin.
However, scientists in New Zealand have found a potential solution to this issue. By using gene-editing techniques, they are altering the color of cows’ coats. By modifying pigmentation genes, the researchers lightened the dark, heat-absorbing fur of common dairy cows. While Holstein-Friesian cows typically have white coats with black patches, the genetically altered calves were born with lighter silver-colored fur.
The researchers aim to refine their approach by incorporating DNA from tropical cattle breeds that are naturally more resilient to high temperatures.
4. Overweight Mice Shed Excess Fat
Gene editing may eventually offer a solution to obesity, according to scientists at Harvard University. In August 2020, they unveiled a novel method to combat weight gain in mice: using CRISPR to convert unhealthy white fat cells into energy-burning brown fat cells.
Stiff white fat cells are packed with harmful lipids that accumulate within the body. Too much white fat can lead to diabetes. On the other hand, brown fat cells are far healthier. They break down some fat to produce energy and store the remaining fat in a more compact form.
The team at Harvard successfully helped mice shed weight by altering their DNA. The scientists transformed the white fat cells, modifying them to behave like the healthier brown fat. They focused on UCP1, a protein present in brown fat that converts chemical energy into heat.
Over the course of the twelve-week study, the mice with white fat cells gained weight, while the gene-edited mice found it much harder to put on pounds. Some even suggest that the gene-editing process helped the mice prevent diabetes.
Scientists believe this method could eventually be developed into a treatment for obesity, although human trials are still a long way off.
3. Gene Editing Takes a Stand Against Superbugs
Antibiotic-resistant superbugs represent an impending global disaster. Harmful pathogens that were once easily treated with penicillin are rapidly gaining resistance to antibiotics. Without swift development of new antibiotics, we could face up to 10 million deaths annually by 2050 due to these dangerous germs.
However, there is a glimmer of hope. Researchers at the University of Manchester have discovered a new method of producing antibiotics using CRISPR gene editing. By combining advanced biological techniques, the team created a unique antibiotic known as malonomycin. This innovative approach may help scientists develop new antibiotics that are more effective against highly resistant superbugs.
“We are now hopeful that our discoveries may lead to the development of new antibiotics,” said Jason Micklefield, the study’s lead researcher. “Additionally, we may find new ways to produce antibiotics, which are urgently needed to fight the emerging drug-resistant pathogens.”
2. Genetically Modified Moths Tackle New York's Pest Crisis
In January 2020, officials in New York State released swarms of genetically modified male moths to help control pests. Young female diamondback moths are particularly destructive, causing significant damage to crops. Despite their brief lifespan, their larvae devour large amounts of crops such as kale, cabbage, and oilseed rape, resulting in around $5 billion in damages each year.
Normally, a pest like the diamondback moth would be tackled with pesticides, but these pests have become highly resistant. In response, Oxitec, a UK-based biotech company, has developed genetically modified male moths to combat these young pests.
Scientists inserted a gene into the male moths that causes newly-hatched larvae to die, but it only affects the females. This means the harmful female larvae will not survive to cause damage. The male larvae will mate with wild females, passing the deadly gene to their offspring. Over several generations, Oxitec predicts the lethal gene will eventually disappear.
1. Researchers Discover a Cure for Mice's Hearing Loss
In 2019, scientists from Harvard Medical School and Boston Children’s Hospital unveiled an innovative treatment for hearing loss in mice, with the potential to be used in humans in the future.
The Beethoven mice, named after the famous German composer who began losing his hearing in his twenties, suffer from a genetic mutation that causes progressive hearing loss and eventual deafness, a condition that also affects humans.
The hearing loss in the mice is triggered by a slight change in their DNA. By leveraging advanced biological techniques, researchers can isolate the defective gene without affecting any other healthy ones. This allows them to reverse the Beethoven mice’s deafness while avoiding any unintended side effects.
However, the researchers caution the public not to get ahead of themselves. There is still a long road of research ahead before this therapy can be applied to humans. Jeffrey Holt from Harvard stated, “We believe our work opens the door toward a hyper-targeted way to treat an array of genetic disorders that arise from one defective copy of a gene. This truly is precision medicine.”
