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Tag: Gene-editing

  • A Gene Editing Therapy Cut Cholesterol Levels by Half

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    In a step toward the wider use of gene editing, a treatment that uses Crispr successfully slashed high cholesterol levels in a small number of people.

    In a trial conducted by Swiss biotech company Crispr Therapeutics, 15 participants received a one-time infusion meant to switch off a gene in the liver called ANGPTL3. Though rare, some people are born with a mutation in this gene that protects against heart disease with no apparent adverse consequences.

    The highest dose tested in the trial reduced both “bad” LDL cholesterol and triglycerides by an average of 50 percent within two weeks after treatment. The effects lasted at least 60 days, the length of the trial. The results were presented today at the American Heart Association’s annual meeting and published in The New England Journal of Medicine.

    The Nobel Prize–winning Crispr technology has mostly been used to address rare diseases, but these latest findings, while early, add to the evidence that the DNA-editing tool could be used to treat common conditions as well.

    “This will probably be one of the biggest moments in the arc of Crispr’s development in medicine,” Samarth Kulkarni, CEO of Crispr Therapeutics, tells WIRED. The company is behind the only approved gene-editing treatment on the market, Casgevy, which treats sickle cell disease and beta thalassemia.

    The American Heart Association estimates that about a quarter of adults in the US have elevated LDL levels. A similar number have high triglycerides. LDL cholesterol is the waxy substance in the blood that can clog and harden arteries over time. Triglycerides, meanwhile, are the most common type of fat found in the body. High levels of both raise the risk of heart attack and stroke.

    The Phase I trial was conducted in the UK, Australia, and New Zealand between June 2024 and August 2025. Participants were between the ages of 31 and 68 and had uncontrolled levels of LDL cholesterol and triglycerides. The trial tested five different doses of the Crispr infusion, which took about two and a half hours on average to administer.

    “These are very sick people,” says Steven Nissen, senior author and chief academic officer of the Heart, Vascular and Thoracic Institute at Cleveland Clinic, which independently confirmed the trial’s results. “The tragedy of this disease is not just that people die young, but some of them will have a heart attack, and their lives are never the same again. They don’t get back to work, they develop heart failure.”

    One trial participant, a 51-year-old man, died six months after receiving the lowest dose of the treatment, which was not associated with a lowering of cholesterol and triglycerides. The death was related to his existing heart disease, not the experimental Crispr treatment. The man had a rare, inherited genetic form of high cholesterol and previously had several procedures to improve blood flow to his heart.

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    Emily Mullin

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  • A New Startup Wants to Edit Human Embryos

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    In 2018, Chinese scientist He Jiankui shocked the world when he revealed that he had created the first gene-edited babies. Using Crispr, he tweaked the genes of three human embryos in an attempt to make them immune to HIV and used the embryos to start pregnancies.

    The backlash against He was immediate. Scientists said the technology was too new to be used for human reproduction and that the DNA change amounted to genetic enhancement. The Chinese government charged him with “illegal medical practices,” and he served a three-year prison sentence.

    Now, a New York–based startup called Manhattan Genomics is reviving the debate around gene-edited babies. Its stated goal is to end genetic disease and alleviate human suffering by fixing harmful mutations at the embryo stage. The company has announced a group of “scientific contributors” that includes a prominent in vitro fertilization doctor, a data scientist who worked for de-extinction company Colossal Biosciences, and two reproductive biologists from a major primate research center. A scientist who pioneered a technique to make embryos using DNA from three people is also involved.

    “I like to take on challenges when I see them,” says cofounder Cathy Tie, a former Thiel fellow who left college at 18 to start her first company, Ranomics, a genomics screening service. As Tie sees it, that challenge is to make the idea of human embryo editing more acceptable in society.

    The idea of editing human embryos is tantalizing, because any changes made to the reproductive cells are heritable. Snip out a disease-causing mutation in an embryo and it would be deleted from future generations as well. But gene-editing technology also has the potential to cause unintended “off-target” effects. Edit the wrong gene by mistake and it could give rise to cancer, for instance. Those mistakes would also be passed down to any future children.

    While newer forms of gene editing are more precise, there are still ethical issues to contend with. The prospect of being able to manipulate the DNA of a human embryo has raised fears of a new kind of eugenics, where parents with the means to do so could make “designer babies” with traits that they select.

    Tie says the goal of Manhattan Genomics—originally called the Manhattan Project when the company first launched in August—is disease correction, not enhancement. Unlike the original Manhattan Project, a secretive US government program during World War II that produced the first nuclear weapons, Tie says her venture will operate openly and transparently. “We’re revolutionizing medicine, and this technology is definitely very powerful. That’s what I think is the commonality here with manipulating the nucleus of the atom and manipulating the nucleus of the cell,” she says.

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    Emily Mullin

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  • Scientists create rechargeable, multicolored, glow-in-the-dark succulents

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    Glow-in-the-dark plants bright enough to light up streets at night may sound like the stuff of science fiction or fantasy.But scientists have already made plants that emit a greenish glow. They are even commercially available in the United States.A group of Chinese researchers has just gone even further, creating what they say are the first multicolored and brightest-ever luminescent plants.”Picture the world of Avatar, where glowing plants light up an entire ecosystem,” biologist Shuting Liu, a researcher at South China Agricultural University in Guangzhou and co-author of the study published Aug. 27 in the journal Matter, said in a statement.”We wanted to make that vision possible using materials we already work with in the lab. Imagine glowing trees replacing streetlights,” she added.To make the plants glow, Liu and her fellow researchers injected the leaves of the succulent Echeveria “Mebina” with strontium aluminate, a material often used in glow-in-the-dark toys that absorbs light and gradually releases it over time.This method marks a departure from the traditional gene-editing technique that scientists use to achieve this effect, following a model pioneered by a team at the Massachusetts Institute of Technology.Injecting a plant with nanoparticles instead of editing its genes allowed the researchers to create plants that glow red, blue, and green. Normally, constrained by the plant’s natural color, scientists can only create a green glow.”Gene editing is an excellent approach,” Liu told CNN in an email Tuesday, but added: “We were particularly inspired by inorganic afterglow materials that can be ‘charged’ by light and then release it slowly as afterglow, as well as by prior efforts on glowing plants that hinted at plant-based lighting — even concepts like plant streetlights.””Our goal was therefore to integrate multicolor, long-afterglow materials with plants to move beyond the usual color limits of plant luminescence and provide a photosynthesis-independent way for plants to store and release light — essentially, a light charged, living plant lamp,” she added.The research team attempted to show the practical application of their idea by constructing a green wall made of 56 plants that produced enough light to see text, images and a person located up to 10 centimeters (4 inches) away, according to the study.Once injected and placed under direct sunlight for a couple of minutes, the plants continued to glow for up to two hours.While the brightness of the afterglow gradually weakened during that time period, “plants can be recharged repeatedly by exposure to sunlight,” Liu said. The sun replenishes the plants’ stored energy, “allowing the plants to continue glowing after the sunlight is removed.”The plants maintain the ability to emit the afterglow effect 25 days after treatment, Liu said, and older leaves injected with the afterglow particles continue to emit light under UV stimulation “even after wilting.”While strontium aluminate can readily decompose in plants, posing harm to plant tissue, Liu said, the scientists developed a chemical coating for the material that acts as a protective barrier.The researchers said in the paper that they see their findings as highlighting “the potential of luminescent plants as sustainable and efficient lighting systems, capable of harvesting sunlight during the day and emitting light at night.”However, other scientists are skeptical about the practicality. “I like the paper, it’s fun, but I think it’s a little beyond current technology, and it might be beyond what plants can bear,” biochemist John Carr, a professor of plant sciences at the University of Cambridge, who was not involved in the study, told CNN.”Because of the limited amount of energy that these plants can emit, I don’t really see them as streetlights anytime soon,” he added.Liu acknowledged that the plants “are still far from providing functional illumination, as their luminescence intensity remains too weak for practical lighting applications. Additionally, the safety assessment of afterglow particles for both plants and animals is still ongoing.”She said the luminescent plants currently “can primarily serve as decorative display pieces or ornamental night lights.”However, Liu added, “Looking ahead, if we can significantly enhance the brightness and extend the duration of luminescence — and once safety is conclusively demonstrated — we could envision gardens or public spaces being softly illuminated at night by glowing plants.”

    Glow-in-the-dark plants bright enough to light up streets at night may sound like the stuff of science fiction or fantasy.

    But scientists have already made plants that emit a greenish glow. They are even commercially available in the United States.

    A group of Chinese researchers has just gone even further, creating what they say are the first multicolored and brightest-ever luminescent plants.

    “Picture the world of Avatar, where glowing plants light up an entire ecosystem,” biologist Shuting Liu, a researcher at South China Agricultural University in Guangzhou and co-author of the study published Aug. 27 in the journal Matter, said in a statement.

    “We wanted to make that vision possible using materials we already work with in the lab. Imagine glowing trees replacing streetlights,” she added.

    To make the plants glow, Liu and her fellow researchers injected the leaves of the succulent Echeveria “Mebina” with strontium aluminate, a material often used in glow-in-the-dark toys that absorbs light and gradually releases it over time.

    This method marks a departure from the traditional gene-editing technique that scientists use to achieve this effect, following a model pioneered by a team at the Massachusetts Institute of Technology.

    Injecting a plant with nanoparticles instead of editing its genes allowed the researchers to create plants that glow red, blue, and green. Normally, constrained by the plant’s natural color, scientists can only create a green glow.

    “Gene editing is an excellent approach,” Liu told CNN in an email Tuesday, but added: “We were particularly inspired by inorganic afterglow materials that can be ‘charged’ by light and then release it slowly as afterglow, as well as by prior efforts on glowing plants that hinted at plant-based lighting — even concepts like plant streetlights.”

    “Our goal was therefore to integrate multicolor, long-afterglow materials with plants to move beyond the usual color limits of plant luminescence and provide a photosynthesis-independent way for plants to store and release light — essentially, a light charged, living plant lamp,” she added.

    The research team attempted to show the practical application of their idea by constructing a green wall made of 56 plants that produced enough light to see text, images and a person located up to 10 centimeters (4 inches) away, according to the study.

    Once injected and placed under direct sunlight for a couple of minutes, the plants continued to glow for up to two hours.

    While the brightness of the afterglow gradually weakened during that time period, “plants can be recharged repeatedly by exposure to sunlight,” Liu said. The sun replenishes the plants’ stored energy, “allowing the plants to continue glowing after the sunlight is removed.”

    The plants maintain the ability to emit the afterglow effect 25 days after treatment, Liu said, and older leaves injected with the afterglow particles continue to emit light under UV stimulation “even after wilting.”

    While strontium aluminate can readily decompose in plants, posing harm to plant tissue, Liu said, the scientists developed a chemical coating for the material that acts as a protective barrier.

    The researchers said in the paper that they see their findings as highlighting “the potential of luminescent plants as sustainable and efficient lighting systems, capable of harvesting sunlight during the day and emitting light at night.”

    However, other scientists are skeptical about the practicality. “I like the paper, it’s fun, but I think it’s a little beyond current technology, and it might be beyond what plants can bear,” biochemist John Carr, a professor of plant sciences at the University of Cambridge, who was not involved in the study, told CNN.

    “Because of the limited amount of energy that these plants can emit, I don’t really see them as streetlights anytime soon,” he added.

    Liu acknowledged that the plants “are still far from providing functional illumination, as their luminescence intensity remains too weak for practical lighting applications. Additionally, the safety assessment of afterglow particles for both plants and animals is still ongoing.”

    She said the luminescent plants currently “can primarily serve as decorative display pieces or ornamental night lights.”

    However, Liu added, “Looking ahead, if we can significantly enhance the brightness and extend the duration of luminescence — and once safety is conclusively demonstrated — we could envision gardens or public spaces being softly illuminated at night by glowing plants.”

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  • He Got a Pig Kidney Transplant. Now Doctors Need to Keep It Working

    He Got a Pig Kidney Transplant. Now Doctors Need to Keep It Working

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    Other than rejection of the organ, one of the most common transplant complications is infection. Doctors have to strike a balance when prescribing immunosuppressive drugs: too low a dose can lead to rejection, while too much can make a patient vulnerable to infection. Immunosuppressants are powerful drugs that can cause a range of side effects, including fatigue, nausea, and vomiting.

    Despite the deaths of the two pig heart recipients, Riella is optimistic about Slayman’s transplant. For one, he says, Slayman was relatively healthy when he underwent the surgery. He qualified for a human kidney but because of his rare blood type he would likely need to wait six to seven years to get one. The two individuals who received pig heart transplants were so ill that they didn’t qualify for a human organ.

    In addition to close monitoring and traditional immunosuppressants, Slayman’s medical team is treating him with an experimental drug called tegoprubart, developed by Eledon Pharmaceuticals of Irvine, California. Given every three weeks via an IV, tegoprubart blocks crosstalk between two key immune cells in the body, T cells and B cells, which helps suppress the immune response against the donor organ. The drug has been used in monkeys that have received gene-edited pig organs.

    Photograph: Massachusetts General Hospital

    “It’s pretty miraculous this man’s out of the hospital a couple of weeks after putting in a pig kidney,” says Steven Perrin, Eledon’s president and chief scientific officer. “I didn’t think we would be here as quickly as we are.”

    Riella is also hopeful that the 69 genetic alterations made to the pig that supplied the donor organ will help Slayman’s kidney keep functioning. Pig organs aren’t naturally compatible in the human body. The company that supplied the pig, eGenesis, used Crispr to add certain human genes, remove some pig genes, and inactivate latent viruses in the pig genome that could hypothetically infect a human recipient. The pigs are produced using cloning; scientists make the edits to a single pig cell and use that cell to form an embryo. The embryos are cloned and transferred to the womb of a female pig so that her offspring end up with the edits.

    “We hope that this combination will be the secret sauce to getting this kidney to a longer graft survival,” Riella says.

    There’s debate among scientists over how many edits pig organs need to last in people. In the pig heart transplants, researchers used donor animals with 10 edits developed by United Therapeutics subsidiary Revivicor.

    There’s another big difference between this procedure and the heart surgeries: If Slayman’s kidney did stop working, Riella says, he could resume dialysis. The pig heart recipients had no back-up options. He says even if pig organs aren’t a long-term alternative, they could provide a bridge to transplant for patients like Slayman who would otherwise spend years suffering on dialysis.

    “We’ve gotten so many letters, emails, and messages from people volunteering to be candidates for the xenotransplants, even with all the unknowns,” Riella says. “Many of them are struggling so much on dialysis that they’re looking for an alternative.”

    The Mass General team plans to launch a formal clinical trial to transplant edited pig kidneys in more patients. They received special approval from the US Food and Drug Administration for just one procedure. For now, though, their main focus is on keeping Slayman healthy.

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    Emily Mullin

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  • Enhanced stem cell culture boosts genome editing safety

    Enhanced stem cell culture boosts genome editing safety

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    Newswise — Tsukuba, Japan—Hematopoietic stem cells (HSCs) are rare cells found in the bone marrow that produce red blood cells, white blood cells, and platelets. Their correct functioning is indispensable for the growth and health of an organism. Accordingly, defects in the DNA of hematopoietic stem cells (mutations) can cause impaired blood production and severe diseases.

    Gene therapy seeks to treat such types of genetic diseases. A breakthrough technology that has driven the entire field in recent years is gene editing via clustered regularly interspaced palindromic repeats/Cas9 (CRISPR/Cas9). Using this technology, one can modify disease-causing mutations and transplant HSCs with recovered function, potentially curing the disease.

    However, the CRISPR/Cas9 system is not perfect. It only corrects mutations in a small fraction of cells and can introduce new, potentially dangerous mutations into other cells. Therefore, selecting corrected cells before transplantation is crucial.

    In 2019, the research group reported a method of expanding HSCs over a long time period using a polymer-based culture system and cytokines. In response to this problem, the authors have developed a novel culture system using a novel high-molecular-weight polymer. This system facilitates the growth of single HSCs in transplantable cell colonies that achieve high blood-producing capacity after long-time ex vivo culture. After editing a mutation in a murine immune deficiency model, the authors individually grow several hundred HSCs and screen them for clones that contain only the desired edit and are expected to engraft successfully. Using this method, the fraction of successfully corrected HSCs used for transplantation can be increased from 20%-30% to 100% while eliminating potentially dangerous mutations from the graft. We believe that this culture system might contribute to improving the efficiency and safety of genome editing in HSCs.

    ###
    This work was supported by the German Research Foundation (BE 6847/1-1 to H.J.B.), the Japan Society for the Promotion of Science (JSPS; #20K16234 to M.S.J.L., #23K15315 to H.J.B., #21F21108 and #20K21612 to S.Y.), the Kay Kendall Leukaemia Fund (A.C.W.), the Japan Science and Technology Agency (JST; #18071245 to C.C.), and the Japanese Agency for Medical Research and Development (AMED; #21bm0404077h0001 and #21bm0704055h0002 to S.Y.). The D.G.K. laboratory is supported by a Blood Cancer UK Bennett Fellowship (15008), an ERC Starting Grant (ERC-2016-STG-715371), a CR-UK Programme Foundation award (DCRPGF100008), the MRC Mouse Genetics Network Haematopoiesis Cluster (MC_PC_21043), and an MRC-AMED joint award (MR/V005502/1).

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    University of Tsukuba

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  • Super crops are coming: Is Europe ready for a new generation of gene-edited plants?

    Super crops are coming: Is Europe ready for a new generation of gene-edited plants?

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    Brussels is finalizing a law to legalize new gene-editing technologies for crops across the European Union.

    The EU’s ultra-restrictive GMO regulation, which predates newer technologies, sets extremely high hurdles for growing genetically engineered crops and allows EU countries to ban them even after they have been proven to be safe.

    The new law aims to cut red tape and allow easier market access for plants grown with “new genomic techniques” (NGTs), such as CRISPR-Cas9, which target specific genes without necessarily introducing genetic material from outside the breeders’ gene pool.

    The rules are being pushed by multinationals such as Bayer, Syngenta and Corteva, which together control the lion’s share of the plant breeding sector, as well as a host of smaller companies, scientists and farmers’ groups such as Copa-Cogeca.

    They argue that the EU risks falling behind the rest of the world in using new crops with special traits that can make them more nutritious, efficient and better adapted to a changing climate.

    Pitted against them are green lawmakers, environmental advocacy groups, organic and small farmers, and more than 400,000 EU citizens who have signed a petition against deregulating what they call “new GMOs.”

    These groups say the rules will further tighten the grip of the handful of multinationals, allowing them to claim patents on crops that could have been obtained through conventional breeding methods, while threatening non-GM and organic production. They also argue that because NGTs have only been around for just over a decade, questions remain about their safety.

    According to a leaked draft, EU countries will no longer be able to ban the cultivation of NGT crops.

    The law simplifies rules even more for a sub-group of NGT crops that are deemed equivalent to crops obtained by traditional breeding techniques. The obligation to label foods as “GMO” will no longer apply to these “conventional-like” plants, and they won’t be subject to risk assessment by food safety regulators.

    An earlier draft of the law had a carve-out for crops engineered to tolerate herbicides — which would still have been subject to the stricter GMO rules. However, a newer draft no longer makes such a distinction.

    The European Commission is due to unveil the proposed law on gene-edited crops on Wednesday, as part of the latest package of measures under its Green Deal environment and sustainability agenda. This will include a new law on soil health, revisions of the food waste and textiles aspects in the EU Waste Framework Directive, and legislation on seeds and other plant and forest reproductive material.

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    Bartosz Brzezinski and Jakob Hanke Vela

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