The common blood test that predicts how fast Alzheimer’s hits

Insulin resistance detected by routine triglyceride-glucose (TyG) index can flag people with early Alzheimer's who are four times more likely to present rapid cognitive decline, according to new research presented at the European Academy of Neurology (EAN) Congress 2025.¹

Neurologists at the University of Brescia reviewed records for 315 non-diabetic patients with cognitive deficits, including 200 with biologically confirmed Alzheimer's disease. All subjects underwent an assessment of insulin resistance using the TyG index and a clinical follow-up of 3 years. When patients were divided according to TyG index, those in the highest third of the Mild Cognitive Impairment AD subgroup deteriorated far more quickly than their lower-TyG peers, losing >2.5 points on the Mini Mental State Examination per year (hazard ratio 4.08, 95% CI 1.06-15.73). No such link appeared in the non-AD cohort.

"Once mild cognitive impairment is diagnosed, families always ask how fast it will progress," said lead investigator Dr. Bianca Gumina. "Our data show that a simple metabolic marker available in every hospital laboratory can help identify more vulnerable subjects who may be suitable candidates for targeted therapy or specific intervention strategies."

While insulin resistance has been linked to the onset of Alzheimer's disease, its role in how quickly the condition progresses has received less attention. This study aimed to fill that gap by focusing on its impact during the prodromal mild cognitive impairment (MCI) stage, when patients follow highly variable trajectories. The researchers used the TyG index, which offers a low-cost, routinely available surrogate for insulin resistance, to explore whether metabolic dysfunction could help predict the pace of cognitive decline after diagnosis.

In AD specifically, insulin resistance is believed to impair neuronal glucose uptake, promote amyloid accumulation, disrupt the blood-brain barrier, and fuel inflammation - pathways that are less relevant or differently regulated in other neurodegenerative diseases.

"We were surprised to see the effect only in the Alzheimer's spectrum and not in other neurodegenerative diseases," Dr. Gumina noted. "It suggests a disease-specific vulnerability to metabolic stress during the prodromal window, when interventions may still change the trajectory."

The researchers at University of Brescia, led by Professor Padovani and Professor Pilotto, found that high TyG was also associated with blood-brain barrier disruption and cardiovascular risk factors, yet it showed no interaction with the APOE ε4 genotype, indicating that metabolic and genetic risks may act through distinct pathways.²

Identifying high-TyG patients could refine enrolment for anti-amyloid or anti-tau trials and prompt earlier lifestyle or pharmacological measures to improve insulin sensitivity. The researchers are currently investigating whether TyG levels also track with neuroimaging biomarkers to aid earlier detection and stratification.

"If targeting metabolism can delay progression, we will have a readily modifiable target that works alongside emerging disease-modifying drugs," concluded Dr. Gumina.

References:

1. Gumina B., Galli A., Tolassi C. et al. The Triglyceride-Glucose Index as Predictor of Cognitive Decline in Alzheimer's Spectrum Disorders. Presented at the European Academy of Neurology (EAN) Congress 2025; 23 June 2025; Helsinki, Finland.
2. Padovani A., Galli A., Bazzoli E., et al. (2025). The role of insulin resistance and APOE genotype on blood-brain barrier integrity in Alzheimer's disease. Alzheimer's & Dementia. Advance online publication. https://doi.org/10.1002/alz.14556

Courtesy:

Beyond. "The common blood test that predicts how fast Alzheimer’s hits." ScienceDaily. ScienceDaily, 22 June 2025. <www.sciencedaily.com/releases/2025/06/250622224303.htm>. 

 

 

Artificial intelligence isn’t hurting workers—It might be helping

As artificial intelligence reshapes workplaces worldwide, a new study provides early evidence suggesting AI exposure has not, thus far, caused widespread harm to workers' mental health or job satisfaction. In fact, the data reveals that AI may even be linked to modest improvements in worker physical health, particularly among employees with less than a college degree.

But the authors caution: It is way too soon to draw definitive conclusions.

The paper, "Artificial Intelligence and the Wellbeing of Workers," published June 23 in Nature: Scientific Reports, uses two decades of longitudinal data from the German Socio-Economic Panel. Using that rich data, the researchers -- Osea Giuntella of the University of Pittsburgh and the National Bureau of Economic Research (NBER), Luca Stella of the University of Milan and the Berlin School of Economics, and Johannes King of the German Ministry of Finance -- explored how workers in AI-exposed occupations have fared in contrast to workers in less-exposed roles.

"Public anxiety about AI is real, but the worst-case scenarios are not inevitable," said Professor Stella, who is also affiliated with independent European bodies the Center for Economic Studies (CESifo) and the Institute for Labor Economics (IZA). "So far, we find little evidence that AI adoption has undermined workers' well-being on average. If anything, physical health seems to have slightly improved, likely due to declining job physical intensity and overall job risk in some of the AI-exposed occupations."

Yet the study also highlights reasons for caution.

The analysis relies primarily on a task-based measure of AI exposure -- considered more objective -- but alternative estimates based on self-reported exposure reveal small negative effects on job and life satisfaction. In addition, the sample excludes younger workers and only covers the early phases of AI diffusion in Germany.

"We may simply be too early in the AI adoption curve to observe its full effects," Stella emphasized. "AI's impact could evolve dramatically as technologies advance, penetrate more sectors, and alter work at a deeper level."

Key findings from the study include:

• No significant average effects of AI exposure on job satisfaction, life satisfaction, or mental health.
• Small improvements in self-rated physical health and health satisfaction, especially among lower-educated workers.
• Evidence of reduced physical job intensity, suggesting that AI may alleviate physically demanding tasks.
• A modest decline in weekly working hours, without significant changes in income or employment rates.
• Self-reported AI exposure suggests small but negative effects on subjective well-being, reinforcing the need for more granular future research.

• Due to the data supply, the study focuses on Germany -- a country with strong labor protections and a gradual pace of AI adoption. The co-authors noted that outcomes may differ in more flexible labor markets or among younger cohorts entering increasingly AI-saturated workplaces.

  "This research is an early snapshot, not the final word," said Pitt's Giuntella, who previously conducted significant research into the effect of robotics on households and labor, and on types of workers. "As AI adoption accelerates, continued monitoring of its broader impacts on work and health is essential. Technology alone doesn't determine outcomes -- institutions and policies will decide whether AI enhances or erodes the conditions of work."

   

  Journal Reference:

• Osea Giuntella, Johannes Konig, Luca Stella. Artificial intelligence and the wellbeing of workers. Scientific Reports, 2025; 15 (1) DOI: 10.1038/s41598-025-98241-3 

Courtesy:

University of Pittsburgh. "Artificial intelligence isn’t hurting workers—It might be helping." ScienceDaily. ScienceDaily, 23 June 2025. <www.sciencedaily.com/eleases/2025/06/250623072753.htm>.

 

Recycled plastic is a toxic cocktail: Over 80 chemicals found in a single pellet

A single pellet of recycled plastic can contain over 80 different chemicals. A new study with researchers from University of Gothenburg and Leipzig shows that recycled polyethylene plastic can leach chemicals into water causing impacts in the hormone systems and lipid metabolism of zebrafish larvae.

The plastic pollution crisis has reached global levels, threatening both planetary and human health, and recycling is proposed as one of the solutions to the plastics pollution crisis. However, as plastics contain thousands of chemical additives and other substances that can be toxic, and these are almost never declared, hazardous chemicals can indiscriminately end up in recycled products.

Increasing gene expressions

In a new study, researchers bought plastic pellets recycled from polyethylene plastic from different parts of the world and let the pellets soak in water for 48 hours. After which zebrafish larvae were exposed to the water for five days. The experimental results show increases in gene expression relating to lipid metabolism, adipogenesis, and endocrine regulation in the larvae.

"These short leaching times and exposure times are yet another indicator of the risks that chemicals in plastics pose to living organisms. The impacts that we measured show that these exposures have the potential to change the physiology and health of the fish," says Azora König Kardgar, lead author and researcher in ecotoxicology at the University of Gothenburg.

"Never full knowledge"

Previous research has shown similar effects to humans, including threats to reproductive health and obesity, from exposure to toxic chemicals in plastics. Some chemicals used as additives in plastics and substances that contaminate plastics are known to disturb hormones, with potential impacts on fertility, child development, links to certain cancers, and metabolic disorders including obesity and diabetes.

"This is the main obstacle with the idea of recycling plastic. We never have full knowledge of what chemicals will end up in an item made of recycled plastic. And there is also a significant risk of chemical mixing events occuring, which render the recycled plastic toxic," says Bethanie Carney Almroth, professor at the University of Gothenburg and principal investigator on the project.

Different chemicals

Apart from the study on the impact that recycled plastics have on zebra fish larvae, the researcher also conducted a chemical analysis of the chemicals leaching from the plastic pellets to the water. And they found a lot of different chemical compounds, but the mixture altered between different samples of pellets.

"We identified common plastics chemicals, including UV-stabilizers and plasticizers, as well as chemicals that are not used as plastics additives, including pesticides, pharmaceuticals and biocides. These may have contaminated the plastics during their first use phase, prior to becoming waste and being recycled. This is further evidence of the complicated issue of plastics waste flows, and of toxic chemicals contaminating recycled plastics," says Eric Carmona, researcher at Department of Exposure Science, Helmholtz Centre for Environmental Research in Leipzig.

"Ban hazardous chemicals"

Representatives from the nations of the world are preparing to head to Geneva, Switzerland, in August, for what is planned to be the final negotiating meeting for a Global Plastics Treaty at the Intergovernmental Negotiating Committee under the United Nations Environmental Program. The authors of the work stress that negotiators and decision-makers must include provisions to ban or reduce hazardous chemicals in plastics, and to increase transparency and reporting along plastics value chains. Plastics cannot be recycled in a safe and sustainable manner if hazardous chemicals are not addressed.

"This work clearly demonstrates the need to address toxic chemicals in plastics materials and products, across their life cycle," says Professor Bethanie Carney Almroth. "We cannot safely produce and use recycled plastics if we cannot trace chemicals throughout production, use and waste phases."

Facts: Polyethylene (PE)

Polyethylene, abbreviated PE, is a type of plastic used in a lot of packaging materials like bottle caps, plastic bags, agricultural mulch films, insulation for wiring and cables, pipes, ropes, toys and household items. It is the most widely produced and used polymer. On plastic products made of polyethylene, the number in the recycling code is either 2 or 4.

ournal Reference:

1. Azora K&ouml;nig Kardgar, Eric Carmona, Therese M. Karlsson, Sara Brosch&eacute;, Bethanie Carney Almroth. Effects of leachates from black recycled polyethylene plastics on mRNA expression of genes involved in adipogenesis and endocrine pathways in zebrafish embryos. Journal of Hazardous Materials, 2025; 495: 138946 DOI: 10.1016/j.jhazmat.2025.138946 

Courtesy:

University of Gothenburg. "Recycled plastic is a toxic cocktail: Over 80 chemicals found in a single pellet." ScienceDaily. ScienceDaily, 23 June 2025. <www.sciencedaily.com/releases/2025/06/250623072802.htm>. 

 

 

DNA floating in the air tracks wildlife, viruses -- even drugs

Dublin is known as a city where you can enjoy a few pints of Guiness, get a warm welcome from the locals and hear lively traditional music drifting out of pubs and into the city air.

But it's not just music floating on the breeze. The air of Dublin also contains cannabis, poppy, even magic mushrooms -- at least their DNA.

That's according to a new study that reveals the power of DNA, vacuumed up from the air, which can track everything from elusive bobcats to illicit drugs.

"The level of information that's available in environmental DNA is such that we're only starting to consider what the potential applications can be, from humans, to wildlife to other species that have implications for human health," said David Duffy, Ph.D., a professor of wildlife disease genomics at the University of Florida and lead author of a new study showing the widespread utility of DNA vacuumed from the air.

Housed at UF's Whitney Laboratory for Marine Bioscience, Duffy's lab developed new methods for deciphering environmental DNA, also known as eDNA, to study sea turtle genetics. They've expanded the tools to study every species -- including humans -- from DNA captured in environmental samples like water, soil and sand.

But these errant strands of DNA do not just settle into muddy soil or flow along rivers. The air itself is infused with genetic material. A simple air filter running for hours, days or weeks can pick up signs of nearly every species that grows or wanders nearby.

"When we started, it seemed like it would be hard to get intact large fragments of DNA from the air. But that's not the case. We're actually finding a lot of informative DNA," Duffy said. "That means you can study species without directly having to disturb them, without ever having to see them. It opens up huge possibilities to study all the species in an area simultaneously, from microbes and viruses all the way up to vertebrates like bobcats and humans, and everything in between."

As a proof of concept, the researchers showed that they could pick up signs of hundreds of different human pathogens from the Dublin air, including viruses and bacteria. Such surveillance could help scientists track emerging diseases. The same method can track common allergens, like peanut or pollen, more precisely than is currently possible, the scientists discovered.

In another test of the power of eDNA, Duffy's lab was also able to identify the origin of bobcats and spiders whose DNA was hoovered up from air in a Florida forest. With little more than an air filter, scientists could track endangered species and identify where they came from, all without having to lay eyes on skittish animals or root around forest floors for scat samples. When trying to save and conserve wildlife, knowing where an animal originates from can be as important as knowing where it currently is.

This powerful analysis was paired with impressive speed and efficiency. The team demonstrated that a single researcher could process DNA for every species in as little as a day using compact, affordable equipment, and software hosted in the cloud. That quick turnaround is orders of magnitude faster than would have been possible just a few years ago and opens up advanced environmental studies to more scientists around the world. The same tools can potentially identify sensitive human genetic data, which is why Duffy and his collaborators have called for ethical guardrails for the rapidly developing field of eDNA.

"It seems like science fiction, but it's becoming science fact," Duffy said. "The technology is finally matching the scale of environmental problems."

Journal Reference:

1. Orestis Nousias, Mark McCauley, Maximilian R. Stammnitz, Jessica A. Farrell, Samantha A. Koda, Victoria Summers, Catherine B. Eastman, Fiona G. Duffy, Isabelle J. Duffy, Jenny Whilde, David J. Duffy. Shotgun sequencing of airborne eDNA achieves rapid assessment of whole biomes, population genetics and genomic variation. Nature Ecology & Evolution, 2025; DOI: 10.1038/s41559-025-02711-w    

 Citation:

University of Florida. "DNA floating in the air tracks wildlife, viruses -- even drugs." ScienceDaily. ScienceDaily, 3 June 2025. <www.sciencedaily.com/releases/2025/06/250603114822.htm>.

Tea, berries, dark chocolate and apples could lead to a longer life span, study shows

New research has found that those who consume a diverse range of foods rich in flavonoids, such as tea, berries, dark chocolate, and apples, could lower their risk of developing serious health conditions and have the potential to live longer.

The study was led by a team of researchers from Queen's University Belfast, Edith Cowan University Perth (ECU), and the Medical University of Vienna and Universitat Wien.

The findings reveal that increasing the diversity of flavonoids within your diet could help prevent the development of health conditions such as type 2 diabetes, cardiovascular disease (CVD), cancer and neurological disease.

Flavonoids are found in plant foods like tea, blueberries, strawberries, oranges, apples, grapes, and even red wine and dark chocolate.

Published in Nature Food, the study tracked over 120,000 participants aging from 40 to 70 years old for over a decade. It is the first study of its kind to suggest that there is a benefit to consuming a wide range of flavonoids beyond that of simply consuming a high quantity.

ECU Research Fellow, first author and co-lead of the study Dr Benjamin Parmenter, made the initial discovery that a flavonoid-diverse diet is good for health.

"Flavonoid intakes of around 500 mg a day was associated with a 16% lower risk of all-cause mortality, as well as a ~10% lower risk of CVD, type 2 diabetes, and respiratory disease. That's roughly the amount of flavonoids that you would consume in two cups of tea."

Dr Parmenter added, however, that those who consumed the widest diversity of flavonoids, had an even lower risk of these diseases, even when consuming the same total amount. For example, instead of just drinking tea, it's better to eat a range of flavonoid-rich foods to make up your intake, because different flavonoids come from different foods.

"We have known for some time that higher intakes of dietary flavonoids, powerful bioactives naturally present in many foods and drinks, can reduce the risk of developing heart disease, type 2 diabetes, and neurological conditions like Parkinson's," study co-lead Professor Aedín Cassidy from the Co-Centre for Sustainable Food Systems and Institute for Global Food Security at Queen's said.

"We also know from lab data and clinical studies that different flavonoids work in different ways, some improve blood pressure, others help with cholesterol levels and decrease inflammation. This study is significant as the results indicate that consuming a higher quantity and wider diversity has the potential to lead to a greater reduction in ill health than just a single source."

Professor Tilman Kuhn from the Medical University of Vienna, Universitat Wien and Queen's University Belfast was also a co-lead author, noted that the importance of diversity of flavonoid intake has never been investigated until now, making this study very significant as the findings align with popular claims that eating colourful foods are invaluable to maintain good health.

"Eating fruits and vegetables in a variety of colours, including those rich in flavonoids, means you're more likely to get the vitamins and nutrients you need to sustain a healthier lifestyle," he said.

The first-ever dietary guidelines for flavonoids were released recently recommending increasing the consumption of flavonoids to maintain health.

"Our study provides inaugural evidence that we may also need to advise increasing diversity of intake of these compounds for optimal benefits," Dr Parmenter said.

"The results provide a clear public health message, suggesting that simple and achievable dietary swaps, such as drinking more tea and eating more berries and apples for example, can help increase the variety and intake of flavonoid-rich foods, and potentially improve health in the long-term," Professor Cassidy added.

Journal Reference:

1. Benjamin H. Parmenter, Alysha S. Thompson, Nicola P. Bondonno, Amy Jennings, Kevin Murray, Aurora Perez-Cornago, Jonathan M. Hodgson, Anna Tresserra-Rimbau, Tilman Kühn, Aedín Cassidy. High diversity of dietary flavonoid intake is associated with a lower risk of all-cause mortality and major chronic diseases. Nature Food, 2025; DOI: 10.1038/s43016-025-01176-1 

Courtesy:

Edith Cowan University. "Tea, berries, dark chocolate and apples could lead to a longer life span, study shows." ScienceDaily. ScienceDaily, 3 June 2025. <www.sciencedaily.com/releases/2025/06/250603115028.htm>. 

Personalized gene editing saved a baby, but the tech’s future is uncertain

When a baby born in Philadelphia was announced as the first person to get a gene therapy designed just for him, many people hailed the achievement as a starting point to treat virtually any genetic disease.

But there is a long road that researchers and regulators need to pave before other people with genetic disorders can get bespoke gene therapies.

Here’s what you need to know about this personalized therapy and how it may affect gene therapy moving forward.

What led to this pioneering gene editing?

On May 15, doctors and researchers at Children’s Hospital of Philadelphia (CHOP) and colleagues described the personalized gene therapy in the New England Journal of Medicine. The treated child, KJ Muldoon, has a disorder that prevents his liver from converting ammonia from broken-up proteins to urea. Urea is flushed from the body in urine.

KJ’s form of the disease stems from a mutation in both copies of his CPS1 gene. That gene contains instructions for building an enzyme called carbamoyl-phosphate synthetase 1 that is important in the urea cycle — the conversion of ammonia to urea. Without the enzyme, ammonia levels shoot up and can cause brain and nerve damage and death. This deficiency affects about 1 in 1.3 million people, about half of whom die in early infancy. Low protein diets, medications that help lower ammonia levels and ultimately liver transplants are used to treat the condition, though these measures may not entirely cure the disorder.

KJ was born prematurely in August and was too small for a liver transplant. Medications and extremely low protein diets helped keep ammonia levels in his blood down. But the levels often spiked, and doctors worried he could be left with permanent brain damage or die.

Cardiologist Kiran Musunuru of the University of Pennsylvania Perelman School of Medicine and pediatrician and medical geneticist Rebecca Ahrens-Nicklas of CHOP had already been practicing for such a scenario. They quickly assembled a coalition of academic and industry scientists to manufacture the gene therapy and make sure it was safe to give to a person, Musunuru said in a news briefing May 12.

The team also applied to the U.S. Food and Drug Administration for permission to treat the baby. The FDA recognized that “KJ was very, very sick and there wasn’t time for business as usual,” Musunuru said. The agency approved the treatment within one week of getting the application.

KJ has gotten three infusions of his personalized gene therapy. He isn’t cured — it’s not known whether all the cells in his liver have the corrective edits. Even some patients with liver transplants can have ammonia spikes after infections. But KJ can eat more protein and needs much less medication to keep his ammonia levels in check, Ahrens-Nicklas said. 

What is the personalized gene therapy?

KJ’s gene therapy is based on CRISPR, a targeted gene-editing system which is being developed to treat cancers and a wide variety of genetic diseases. This child got a molecular pencil version of CRISPR called a base editor. That editor chemically erased a mutation in KJ’s broken CPS1 gene and wrote in the correct DNA letter, or base. Base editors are being used as possible treatments for high cholesterol and other conditions. (Musunuru is a founder of the company developing the cholesterol gene therapy.)

This therapy started with messenger RNA, or mRNA, instructions for making the base editor. Messenger RNA is a go-between molecule, a copy of DNA instructions for building a protein. The mRNA is read by cellular machinery and used to produce the protein, which then carries out a particular job. In this case, making the base editor that would correct the typo in KJ’s gene.

The researchers encased mRNA instructions for making the base editor in a lipid nanoparticle, which is “basically like a soap bubble,” says Petros Giannikopoulos, a molecular pathologist at the University of California, Berkeley’s Innovative Genomics Institute. Giannikopoulos was involved in testing KJ’s base editor to make sure it didn’t cause unintended changes elsewhere in his DNA. COVID mRNA vaccines made by Moderna and Pfizer-BioNTech use similar lipid nanoparticle technology to deliver mRNA to cells. In KJ’s case, the nanoparticles specifically directed the therapy to the liver.

Fixing a DNA typo

Researchers used a CRISPR base editor to repair a typo in one baby’s gene, in this case changing an incorrect adenine (A) to the correct guanine (G). A guide RNA (orange) shepherds the base editor to the typo. The base editor is a version of a DNA-cutting enzyme called CRISPR/Cas9 that has been altered so that it nicks one of two DNA strands at the typo (therefore, called Cas9 nickase). Tacked on to the Cas9 nickase is another enzyme called a DNA adenine deaminase. It chemically converts the DNA base adenine (A) to inosine (I). Inosine isn’t a usual DNA building block so DNA repair enzymes in a cell convert it to guanine (G), fixing the typo.

J. Hirshfeld

Researchers tested the base editor in lab-grown cells in a dish. They then gave doses of the therapy to crab-eating macaques to test for toxicity. The team also genetically engineered mice to carry KJ’s mutation, then used the base editor to correct the DNA typo. It took only six months to develop and test the therapy.

KJ got his first intravenous infusion containing the therapy in February. He got a very low dose to start with. Two subsequent doses have been higher. The researchers will continue to monitor KJ, and he soon may be able to leave the hospital where he has lived since birth.

What makes KJ’s case special?

“What was very unique about this was that the therapy was manufactured for an individual patient,” Giannikopoulos says. “This child had a specific mutation…. The therapy was designed for that specific mutation. That was why this was so monumental.”

Previous CRISPR and other gene therapies, including one approved by the FDA to treat sickle cell disease and beta-thalassemia, are proactive, he says. That means “we make and preapprove something, and then wait for the patients to come along” whose mutations match the therapy. KJ’s treatment, in contrast, is reactive. “The patient was diagnosed. We sequenced and found the defect in the genome. And then we designed and manufactured and did all the work to target that specific mutation.”

That personalized approach could be used for many people with super rare diseases.

“I don’t think I’m exaggerating when I say that this is the future of medicine,” Musunuru said in the news briefing. “This is a step towards the use of genetic editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments.”

Giannikopoulos agrees. “What happened at CHOP was basically the birth of a new medical subspecialty,” he says.

 

Can every genetic disease be treated this way?

Probably not all of them, Giannikopoulos says. There are more than 7,000 known genetic diseases. He estimates that 15 to 20 percent of those might be fixable using currently available gene-editing technology. Ones that are caused by single letter typos in a single gene might be correctable using a base editor.

Other CRISPR editors, including a very versatile version called a prime editor, potentially can repair many types of mutations, including small deletions. Cambridge, Mass.–based Prime Medicine announced in a news release May 19 that it had successfully treated a person with a different rare disease using prime editing.

Scientists must also be able to easily get the editor where it needs to go. Diseases that affect easily accessible organs may be the most treatable, Giannikopoulos says. Immune system or blood disorders may be fixed by removing stem cells from the bone marrow, editing them in the lab and then returning them to the patient. Other organs such as the skin, eye or liver (as in KJ’s case) are relatively easy to deliver gene therapy to inside the body. Other gene therapies delivered inside the body include an approved treatment for Duchenne muscular dystrophy.

But diseases that affect multiple organs at once or those that affect hard-to-reach organs might not be easily treated. For instance, “we’re not very good at targeting the kidney right now,” Giannikopoulos says.

When to deploy gene therapy also matters. “For some diseases, you might need to intervene, maybe in utero, but we haven’t gotten there yet.”

“You’ve also got to understand the disease well enough to be able to intervene. Because [for] some diseases, we know what the mutation is, but we don’t really understand how the mutation is causing trouble.”

What else is needed for personalized gene therapy to become widespread?

Changes in regulations would probably be necessary, Giannikopoulos says. Currently, gene therapies are usually approved for correcting a specific mutation. But some genes may have hundreds of disease-causing mutations, so only a subset of people with a particular disease may be eligible.

Instead, Giannikopoulos and other researchers argue that the general procedure and materials, including the delivery vehicle and gene editor, should be treated as a platform or umbrella therapy. That umbrella intervention would be tested for safety and efficacy and then could be deployed as needed for a patient’s particular mutation. “Otherwise, if everything needs to be repeated in terms of the safety and efficacy for every one of these tens of thousands or potentially hundreds of thousands of specific mutations, then we’ll never get there,” Giannikopoulos says.

It may also be a struggle to get insurance to pay for expensive one-off gene therapies, he says. Funds have been a common and concerning theme for gene therapy. Even when therapies have been proven to work, companies often don’t have the resources to conduct clinical trials to get FDA approval, or to keep the treatments on the market once they have been approved. For instance, Prime Medicine is no longer developing its just-announced gene therapy.

Drastic cuts in research funding may also hinder early gene therapy development in academic laboratories in the United States.

Standardized playbooks for designing and implementing gene therapies are also needed because many doctors want to treat patients with genetic diseases, but don’t have the know-how, Giannikopoulos says. He and Musunuru are part of the U. S. National Institutes of Health–funded Somatic Cell Genome Editing Consortium that is putting together such playbooks. “That’s going to be really how to scale this, [by] teaching everybody how to fish around the world.”

Questions or comments on this article? E-mail us at feedback@sciencenews.org | Reprints FAQ

 

Citations

K. Musunuru et al. Patient-specific in vivo gene editing to treat a rare genetic disease. New England Journal of Medicine. Published online May 15, 2025. doi: 10.1056/NEJMoa2504747.

 

Couresty: https://www.sciencenews.org/article/personalized-crispr-gene-edit-therapy