Tag Archives: CRISPR

Creating a Bitterness-Free CRISPR Grapefruit: A Breakthrough in Genetic Modification

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Grapefruit with Reduced Bitterness

Grapefruit with Reduced Bitterness in Development

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Have you skipped eating grapefruit due to its bitterness? The new CRISPR gene-edited variety may change your mind. Researchers have discovered that by disabling a specific gene, they can greatly diminish the bitter compounds found in grapefruit.

“The market for grapefruit could significantly expand,” states Nil Karmi from the Volcano Center in Rishon Lezion, Israel. “Many children avoid grapefruit due to its bitter taste.”

Karmi posits that this innovative approach could also aid the citrus industry amidst the threat of a devastating bacterial disease known as citrus greening.Huanglongbing poses significant risks to citrus, but cold-resistant varieties might mitigate these problems. “The insects responsible for spreading the disease cannot survive in regions with cold winters; however, the citrus varieties that can tolerate the cold are often too bitter for consumption,” he explains.

Gene-editing technology opens doors to creating cold-tolerant edible citrus varieties, allowing for cultivation in regions with temperate climates, such as Northern Europe, instead of only subtropical areas like Florida.

Citrus fruits have their distinctive sourness, particularly evident in lemons, but their bitterness stems from various compounds. Previous studies indicate that grapefruit’s bitterness is primarily linked to a compound called naringin, alongside related molecules like neohesperidin and poncitin.

To address this, Karmi’s team utilized CRISPR gene editing on a grapefruit variety to deactivate the genes responsible for producing these three bitter compounds. While grapefruit trees take several years to bear fruit, preliminary tests on the leaves show no presence of naringin, indicating that the fruit will likely be less bitter.

The modified trees also carry “marker genes” that facilitate easy identification of successfully edited plants. However, these marker genes complicate and increase the cost of obtaining permission to sell the fruit in various countries. In places like the United States and Japan, simple gene edits are not classified as genetic modifications, easing the approval process.

The team plans to replicate these gene edits in grapefruit without incorporating marker genes. “It’s a feasible plan, but it requires extensive effort,” adds Elena Plesser, also from the Volcano Center. “The process is quite challenging.”

While research teams globally are exploring similar gene-editing strategies, Karmi believes his group’s advancements are noteworthy.

The researchers are also targeting the same enzymes in cold-tolerant citrus varieties, such as pomelo, whose fruits are currently inedible due to elevated bitterness levels. The goal is to cross-breed these with popular citrus varieties like oranges to maintain cold hardiness while generating delicious, seedless fruit—a process expected to take years.

This gene editing may revolutionize the taste profile of numerous citrus fruits, claims Erin Mulvihill, who has studied naringin at the University of Ottawa, Canada.

Moreover, grapefruit consumption can pose challenges for some medications, particularly statins, as it inhibits liver enzymes responsible for processing these drugs, risking dangerously high drug levels for users. Naringin is a major player in these interactions, but, according to Muribihir, it’s not the sole factor. “To eliminate all grapefruit-drug interactions, multiple gene deletions would be necessary,” he states.

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Source: www.newscientist.com

CRISPR: Revolutionizing Genetic Code Editing – The Most Innovative Idea of the Century

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New Scientist: Your source for the latest in science news and long-form articles from expert journalists covering advancements in science, technology, health, and environmental issues.

“The pain was like being struck by lightning and being hit by a freight train at the same time,” shared Victoria Gray. New Scientist reflects on her journey: “Everything has changed for me now.”

Gray once endured debilitating symptoms of sickle cell disease, but in 2019, she found hope through CRISPR gene editing, a pioneering technology enabling precise modifications of DNA. By 2023, this groundbreaking treatment was officially recognized as the first approved CRISPR therapy.

Currently, hundreds of clinical trials are exploring CRISPR-based therapies. Discover the ongoing trials that signify just the beginning of CRISPR’s potential. This revolutionary tool is poised to treat a wide range of diseases beyond just genetic disorders. For example, a single CRISPR dose may drastically lower cholesterol levels, significantly reducing heart attack and stroke risk.

While still in its infancy regarding safety, there’s optimism that CRISPR could eventually be routinely employed to modify children’s genomes, potentially reducing their risk of common diseases.

Additionally, CRISPR is set to revolutionize agriculture, facilitating the creation of crops and livestock that resist diseases, thrive in warmer climates, and are optimized for human consumption.

Given its transformative capabilities, CRISPR is arguably one of the most groundbreaking innovations of the 21st century. Its strength lies in correcting genetic “misspellings.” This involves precisely positioning the gene-editing tool within the genome, akin to placing a cursor in a lengthy document, before making modifications.

Microbes utilize this genetic editing mechanism in their defense against other microbes. Before 2012, researchers identified various natural gene-editing proteins, each limited to targeting a single location in the genome. Altering the target sequence required redesigning the protein’s DNA-binding section, a process that was time-consuming.

However, scientists discovered that bacteria have developed a diverse range of gene-editing proteins that bind to RNA—a close relative of DNA—allowing faster sequence matching. Producing RNA takes mere days instead of years.

In 2012, Jennifer Doudna and her team at the University of California, Berkeley, along with Emmanuelle Charpentier from the Max Planck Institute for Infection Biology, revealed the mechanics of one such gene-editing protein, CRISPR Cas9. By simply adding a “guide RNA” in a specific format, they could target any desired sequence.

Today, thousands of variants of CRISPR are in use for diverse applications, all relying on guide RNA targeting. This paradigm-shifting technology earned Doudna and Charpentier the Nobel Prize in 2020.

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Source: www.newscientist.com

Gene-Edited Babies: The Future of Genetics, but Not All CRISPR Startups Will Lead the Way

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Babies Crawling in Diapers

Every Baby Has About 100 New Genetic Mutations

Mood board – Mike Watson / Getty Images

Let me share some eye-opening news. Every child embodies genetic experimentation, with nature exhibiting indifference if things don’t go as planned. Our genomes present a complex tapestry shaped by conflicting evolutionary forces, and each of us carries roughly one hundred novel mutations.–Each birth introduces a unique mutation into the genetic pool.

Thus, I anticipate that in the future, gene editing of embryos will become commonplace once humanity confronts various daunting challenges, including climate change. There may come a time when natural conception is perceived as reckless.

Reaching that future is no trivial task. However, if you’ve been following the buzz from the tech community this year, it’s no surprise you feel optimistic. By 2025, we discovered at least three startups focused on creating gene-edited babies.

So, is the dawn of CRISPR on the horizon, or could these startups potentially face backlash?

Preventing Genetic Diseases

Among these startups, Manhattan Genomics and Preventive aim not for enhancement but to avert severe genetic disorders. This noble objective is commendable, but it’s important to note that many of these conditions can already be forestalled through existing screening techniques, such as genetic testing of IVF embryos prior to implantation, a process with a high rate of success.

So why pursue the development of gene-edited embryos, a complex and legally challenging endeavor, when IVF screening already provides a viable solution?

Preventive did not respond to inquiries, but a spokesperson from Manhattan Genomics noted that couples undergoing IVF often don’t have enough viable embryos to choose from. By editing disease-carrying embryos instead of discarding them, the likelihood of having a healthy child increases. The company believes that gene editing could enhance the chances for approximately ten embryos affected by Huntington’s disease and thirty-five embryos affected by sickle cell disease annually for couples using IVF.

However, this translates to a very limited number of births. Approximately one-third of IVF embryos lead to viable births, and this percentage may drop further post-editing. Furthermore, significant challenges accompany this approach. Although CRISPR technology has advanced, there’s still a risk of introducing harmful mutations as unintended consequences.

Moreover, the editing process often fails to initiate or can continue even after the embryo has begun dividing. This results in various genetic alterations within the same embryo, a phenomenon known as mosaicism. The illegal CRISPR children from China come to mind, announced in 2018.

Consequently, it becomes uncertain whether the mutation causing the disease was indeed corrected in the edited embryo and whether any harmful mutations emerged as a result.

Doing It Right

Solutions do exist. For instance, some gene-edited animals have been developed by modifying stem cells and then cloning them once the desired alterations have been confirmed. However, I previously explained that cloned animals often exhibit various health issues and unexpected traits, underscoring the necessity for foundational research and rigorous oversight should this approach be pursued for humans.

We have two strong examples of responsibly introducing embryonic gene editing through mitochondrial donation initiatives in the UK and Australia. Mitochondria are cellular energy producers that contain their own small genomes. Mutated mitochondria can lead to severe health issues if passed down to offspring, but this risk can be mitigated by substituting them with healthy donor mitochondria.

A version of mitochondrial technology emerged in private fertility clinics in the US during the 1990s, during which humanity witnessed the first genetically modified human. Initial attempts led to the banning of this technology in the US.

While mitochondrial donation was previously prohibited in the UK, changes in the law came about following advocacy from patient groups, extensive dialogue, and consultation. It now receives approvals on a trial basis as needed.Australia is pursuing a similar path.

What Is the Real Objective?

This is the ideal framework for introducing new reproductive technologies: transparently, legally, and under independent supervision. Yet, at least two startups are reportedly conducting experiments in countries with laxer gene editing laws.

This does not advance science, as trust in the claims made by private companies acting without regulatory oversight diminishes. Conversely, this approach could prompt a backlash, leading to more countries tightening regulations against gene editing.

For these billionaires – with Preventive’s investors including notable figures like OpenAI’s Sam Altman and Coinbase’s Brian Armstrong – if your genuine intention is to combat severe genetic diseases, investing in nonprofit research organizations could yield significantly greater results.

Or is the ultimate aim to engineer your own child instead of assisting other couples in achieving healthy pregnancies? This is clearly the mission of the third startup, Bootstrap Bio.

In next month’s column, we will explore whether gene editing can truly be utilized to enhance our children.

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Source: www.newscientist.com

A Baby with a Rare Disease Receives the World’s First Personalized CRISPR Gene Therapy

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Baby KJ Rebecca Affles Nicklas and Kiran Musnul after gene editing injection with researchers

Philadelphia Children’s Hospital

A young boy afflicted with a serious genetic disorder is set to be the first recipient of personalized CRISPR gene editing treatments, offering a glimpse into the potential future of medicine.

This groundbreaking event marks the first instance of an individual receiving a gene editing therapy tailored to correct unique mutations contributing to their illness. Rebecca Ahrens-Nicklas explained during a press briefing held at Children’s Hospital in Philadelphia, Pennsylvania, “He is showing early signs of progress,” though she noted that it’s premature to determine the complete effectiveness of the treatment.

Researchers released information promptly, aiming to motivate others, as stated by team member Kiran Musnur at the University of Pennsylvania. “We sincerely hope that demonstrating the feasibility of personalized gene editing therapy for one patient within a few months will encourage additional efforts in this area,” he remarked.

“When I refer to this as the future of medicine, I believe I’m stating a fact,” he emphasized. “This is a crucial step towards employing gene editing therapies to address a range of rare genetic disorders that currently lack viable treatment options.”

KJ inherited mutations on both alleles of a liver enzyme gene known as CPS1. The absence of this enzyme leads to ammonia accumulating in the bloodstream, posing a risk of brain damage during the breakdown of dietary proteins. According to Ahrens-Nicklas, over half of children born with CPS1 deficiency do not survive.

She and Musnur are developing therapies targeting this condition by focusing on the liver, allowing them to rapidly formulate a basic editing therapy that modifies one of KJ’s two CPS1 gene copies.

The team engaged with US regulatory bodies early in the process. “They recognized the exceptional nature of this situation,” Musnur stated. “KJ was critically ill and time was of the essence. Following our official submission to the FDA [Food and Drug Administration] when KJ was six months old, we received approval within just a week.”

KJ underwent initial low-dose treatment at six months in February 2025, followed by higher doses in March and April. He is now able to consume more protein than before, albeit while still taking other medications for his condition.

Ideally, children should receive treatment earlier to mitigate long-term damages linked to conditions like CPS1 deficiency. As reported by New Scientist, Musnur has ambitions to enable gene editing in humans prior to birth one day.

In contrast, other gene editing therapies are designed for broader applications, aiming to work for many individuals irrespective of the specific mutations causing their condition. For instance, the first approved gene editing treatment for sickle cell disease functions by enhancing fetal hemoglobin production, rather than altering the mutations in adult hemoglobin responsible for the disorder. Despite being a “one-size-fits-all” solution, it comes at a price of £1,651,000 per treatment in the UK, as noted by Each treatment course costs £1,651,000.

KJ with his family after treatment

Philadelphia Children’s Hospital

Custom treatments can be significantly more costly. Musnur mentioned that he cannot provide exact figures for KJ’s treatment due to the extensive pro bono work by the involved companies. However, he is optimistic about a decline in costs. “As we enhance our methods, we can anticipate economies of scale, leading to a substantial reduction in prices,” he stated.

One barrier to the development of personalized gene editing therapies has been the regulatory perspective, which previously treated therapies targeting different mutations within the same gene as separate entities. This necessitated restarting the approval process for each mutation individually. However, there’s a growing movement towards a platform approach, allowing broader approvals for treatments targeting various mutations.

“Platform-based methods, like CRISPR gene editing, offer scalable solutions for even the rarest diseases, as exemplified by KJ’s case,” stated Nick Mead from Genetic Alliance UK, a charity that advocates for individuals with rare conditions. “This development finally renders treatment a plausible possibility for countless families.”

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Source: www.newscientist.com

CRISPR gene editing brings us sweeter tomatoes

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Gene editing can make larger tomato varieties sweeter

Paul Maguire/Shutterstock

If you like sweet tomatoes, smaller cherry tomato varieties are the way to go right now. But larger tomato varieties could soon be enhanced for sweetness with the help of CRISPR gene editing.

Jinzhe Zhang of the Chinese Academy of Agricultural Sciences in Beijing said the larger the tomato, the lower the sugar content usually is. Efforts to increase the sweetness of large varieties also had downsides, such as lower yields.

Zhang and colleagues compared different varieties to identify genetic variations that affect sweetness. They discovered two closely related genes called. SlCDPK27 and SlCDPK26 Larger varieties are more active. These genes code for proteins that reduce the levels of sugar-producing enzymes.

When the research team used CRISPR gene editing to disable these genes in a variety called Moneymaker, glucose and fructose levels in the fruit increased by up to 30% without any loss in yield. Taste tests also rated the fruit as sweeter. The only other effect is that the number of species that consumers are likely to prefer will be smaller and smaller.

“We are working with several companies to develop several commercial varieties by knocking out these genes,” Zhang says. “It's still in the early stages.”

In addition to increased sweetness, another potential benefit is that fewer tomatoes are needed to make tomato ketchup with the same sweetness level.

Gene-edited Money Maker tomatoes aren't as sweet as cherry varieties such as Sungold, but they could be made even sweeter, Zhang said. “Many important genes that control sugar are still waiting to be discovered.”

CRISPR-edited tomatoes, which contain high concentrations of a beneficial nutrient called GABA, are already on sale in Japan, the first CRISPR food to be sold, and are sometimes given as seedlings.

Tomatoes were also the first genetically modified food to be sold commercially. Called Flavr Savr, it was sold in paste form in the United States starting in 1994, but was later discontinued. Since last year, purple GM tomatoes rich in anthocyanins have become available in the United States in fruit and seedling form.

Several countries, including Japan and China, have regulations that make it easier to obtain approval for gene-edited crops compared to other forms of genetic modification, except for conventional breeding. approved by china Last year, the first gene-edited crop was created. Soybeans have high levels of oleic acid.

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Source: www.newscientist.com

‘Refining Bridge Editing: A Potential Improvement Over CRISPR for DNA Modification’

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Bridge editing physically links two DNA strands

Visual Science

A powerful DNA-editing mechanism discovered in bacteria has the potential to enable much larger genome modifications than are currently possible with CRISPR-based techniques, but it is not yet clear whether it would work in human cells.

Patrick Shue Researchers at the Ark Institute in California call their new genome editing system the “bridge editing” system because it physically links, or bridges, two strands of DNA. “Using this system, we can modify large parts of the genome,” says Su. Su's team has figured out how bacterial “parasitic” DNA sequences naturally replicate using this system and how it can be applied to genome editing.

“We're excited about the possibility of making much broader genome modifications that go far beyond what we can do today with CRISPR,” he says, “and we see this as an important step toward a broader vision for genome design.”

CRISPR gene editing has revolutionized biology since it was introduced in 2012. It is used for a variety of purposes, and the first CRISPR-based therapeutic was approved last year. However, the basic form of CRISPR, which uses the Cas9 protein, is gene disruption rather than gene editing.

A standard CRISPR Cas9 protein has two parts: one part binds to a guide RNA molecule and looks for DNA that matches a specific section of the guide RNA. Custom guide RNAs are easy to create, so CRISPR Cas9 can be “programmed” to look for any part of the genome.

The second part of CRISPR Cas9 is a cutter that cuts the DNA when Cas9 binds to the target site. Once the cell has repaired the damage, Cas9 cuts it again. This action continues until an error is made during repair, resulting in the intended mutation of the target site.

While being able to mutate specific sites is useful, biologists want to make more precise changes, so they are modifying CRISPR proteins to edit DNA directly rather than relying on cellular repair mechanisms. For example, base editors can change one DNA letter to another without cutting the DNA, while prime editors can convert an extra section of guide RNA into DNA and add it to the target site.

These improved versions of CRISPR have the potential to treat a wide range of diseases, with several clinical trials already underway, but to address some diseases, more sophisticated genome modifications are needed. Many teams around the world are working on ways to achieve this. Some have realized that the mechanism by which genetic parasites cut and paste from one part of the genome to another, called IS110 elements, has potential because, like CRISPR, it is RNA-guided, but Hsu's team is the first to fully understand how it works.

The bridge-editing system consists of a so-called recombinase protein that binds to a guide RNA, such as the CRISPR Cas9 protein. What's unique about this system is that the guide RNA specifies two DNA sequences to seek out, not just one, Hsu's team found.

One sequence specifies the target site in the genome to modify, similar to CRISPR, and the other specifies the DNA to change. Using this system, DNA sequences of virtually any length can be added, deleted, or inverted.

There are already ways to do this, but they typically require multiple steps and leave behind a piece of extra DNA called a scar. “Bridge editing leaves virtually no scar,” Hsu says. “It offers an unprecedented level of control in engineering the genome.”

This means that it could be used for more than just replacing faulty genes, he says: It could also be useful for completely remaking the genomes of plants and animals. “What we want to do is go from inserting individual genes to doing chromosome-scale genome engineering,” Su says.

“The findings reported are certainly exciting and the underlying biology is truly surprising.” Steven Tang Bridge editing is being done at Columbia University in New York, but so far it has only been demonstrated to work in bacterial cells or in test tubes. Tang says it remains to be seen whether and to what extent bridge editing will work in complex cells like humans. But even if bridge editing doesn't work in initial tests in human cells, it may be possible to modify the system to work over time.

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Source: www.newscientist.com

Lab experiment suggests CRISPR can disable and treat HIV

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Electron micrograph of HIV, which currently requires lifelong medication

Scott Kamazin/Alamy Stock Photo

A new way to eradicate HIV from the body could one day become a cure for infection with the virus, but it has not yet been proven effective in humans.

The strategy uses a relatively new genetic technology called CRISPR, which can make cuts in DNA and introduce errors into the viral genetic material within immune cells. “These findings represent a vital advance toward the design of therapeutic strategies,” the researchers said. Elena Herrera Carrillo Researchers from the University of Amsterdam in the Netherlands said in a statement.

HIV infection used to be almost always fatal, but now people with the virus can take drugs that stop the virus from replicating. Therefore, as long as you diligently take your medication every day, you can maintain a nearly normal lifespan.

But when a person is first infected, some of the virus inserts its DNA into immune cells, where it becomes dormant. When you stop taking your HIV medication, this DNA “reawakens” and the virus begins to spread again through your immune system.

For treatment, we need some way to kill the latent virus in the body. Several strategies have been tried, but so far none have been found to work.

The latest approach uses a gene editing system called CRISPR. Originally discovered in bacteria, it targets specific DNA sequences and makes cuts there. By changing the targeted DNA sequence, the system could be applied as a form of gene therapy for many conditions, and such treatments were launched in the US and UK last year as a treatment for sickle cell anemia. first approved.

Several groups are working with CRISPR to target HIV genes as a way to disable the dormant virus. Now, Carrillo and her team have shown that when tested on immune cells in a dish, the CRISPR system can neutralize all viruses and remove them from these cells. The work will be presented at European Conference on Clinical Microbiology and Infectious Diseases Next month in Barcelona, Spain.

Jonathan Stoy Researchers at London’s Francis Crick Institute say the results are encouraging, but the next step is testing in animals and eventually humans to ensure the treatment is available to all people with dormant HIV. The aim is to show that it can reach immune cells, he said. Some of these cells are thought to reside in the bone marrow, but other parts of the body may also be involved, he says. “There is still considerable uncertainty about whether there are other stores in other parts of the body,” he says.

A California company called Excision BioTherapeutics has previously shown that a CRISPR-based approach can: Reduce the amount of latent virus in monkeys infected with a virus similar to HIV.

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Source: www.newscientist.com

CRISPR gene therapy shows promise in treating severe inflammatory conditions

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New treatment cuts the gene for kallikrein, a protein involved in inflammation (illustrated)

BIOSYM TECHNOLOGIES, INC./Science Photo Library

Nine people with a rare genetic disease that causes a life-threatening inflammatory response appear to have been cured after taking part in the first trial of a new version of CRISPR-based gene therapy.

This condition, called hereditary angioedema, causes sudden swelling of tissue that affects parts of the body such as the face and throat, similar to aspects of an allergic reaction, but cannot be treated with anti-allergy drugs.

Ten people who received a one-time gene therapy administered directly into the body saw a 95 per cent reduction in the number of 'swelling attacks' in the first six months after the treatment took effect. . Since then, all but one have had no further seizures for at least a year, although one patient who received the lowest dose had one mild seizure. “This is potentially a cure,” he says Padmalal Gurugama At Cambridge University Hospital in the UK, we worked on a new approach.

Hereditary angioedema is usually caused by mutations in the gene that encodes a protein called C1 inhibitor, which is involved in suppressing inflammation, which is part of the immune response.

People with this condition may experience a sudden buildup of fluid under their skin several times a month, which is painful and can cause suffocation if it gets stuck in the throat. This attack can be caused by a virus, changes in hormone levels, or stress.

Existing drugs that can reverse attacks work by blocking another molecule involved in inflammation called kallikrein, which is made in the liver. Because people can be born without the ability to make kallikrein without adverse effects, the results suggest that it is safe to permanently block kallikrein through gene therapy, Gurgama said.

The new treatment, developed by a company called Intellia Therapeutics in Cambridge, Massachusetts, consists of genetic material designed to cut the kallikrein gene. It is encapsulated in lipid nanoparticles and taken up by liver cells. One person was treated in the UK and nine in New Zealand and the Netherlands.

An unusual feature of this therapy is that it is administered directly to humans, a method also referred to as “in vivo” delivery. “They get one infusion and that's it,” he says. julian gilmore from University College London was not involved in the study. “It's very appealing.”

So far, most other CRISPR-based gene therapies have been administered “outside the body.” This means a more complex and time-consuming procedure of taking some of a person's cells outside the body, changing the cells in a lab, and then reinjecting them.

CRISPR gene therapy is being developed for multiple genetic diseases, with the first treatments recently approved in the UK and US to help patients with two forms of genetic anemia: sickle cell disease and beta-thalassemia. Ta.

The success of the latest trial is “very exciting,” Gilmore said. Development of CRISPR-based treatments for people with various liver-related conditions, called transthyretin amyloidosis. “This technology could be applied to any disease caused by a mutant protein produced exclusively in the liver, where it is desirable to knock down that protein,” he says.

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Source: www.newscientist.com