Latest news with #CPS1
Yahoo
a day ago
- General
- Yahoo
World's first patient treated with CRISPR gene editing therapy at CHOP returns home
The Brief A ten-month-old baby who has been at the Children's Hospital of Philadelphia (CHOP) since birth finally got to go home with his family. KJ Muldoon made headlines around the globe a few weeks ago for being the first person in the world to receive a breakthrough gene editing therapy that is customized to the patient. The family said after KJ received three infusions in February, March and April. Doctors said the results are very promising so far. CLIFTON HEIGHTS, Pa. - An emotional homecoming occurred in Delaware County for a ten-month-old baby named KJ Muldoon who made headlines around the globe just a few weeks ago. The backstory KJ was born on August 1 and diagnosed with a rare metabolic disease called CPS1 that causes ammonia to build up to a toxic level in the body. In February, doctors treated the infant with a breakthrough and historic CRISPR gene editing therapy, making KJ the very first patient in the world to receive this kind of personalized treatment. KJ received additional infusions of the experimental therapy in March and April. Doctors have told his parents the results so far are very promising. What's New "We went through all of the emotions. You're excited, you're nervous, but we're just glad that he's finally able to be home with us," said Nicole Muldoon, KJ's mother. "We've been operating like five plus one for so long and we're excited to be the six of us moving forward." "We're trying to meet all his developmental milestones and kind of see what he's capable of, but he's already shown us how special he is and I think we're in for a treat." KJ's family and friends hung up welcome home signs and colorful balloons as they waited in anticipation for his homecoming in Clifton Heights. "It's just been a really long fight for him," said Dee Aaron, KJ's grandmother. "Miracles do happen, and it really is a miracle. We didn't think he'd be here." "We're so happy you're home big guy," said Cathy Franklin, KJ's great-grandmother. "He's beautiful, and his parents have been remarkable just remarkable. Stayed so strong and we just prayed for ten months. Here he is!" The staff at CHOP dressed KJ up in a graduation cap and gown for his send-off, and they sent him out the hospital doors in great numbers, cheering on his health and recovery. Once the family made it out of the hospital, police from Upper Darby and Radnor Township escorted the family from CHOP all the way to Clifton Heights. The nonprofit The Delco Group helped arrange the special police escort. "We made one phone call yesterday with Ken Piree from Radnor right to Upper Darby Township. It was just within seconds and that's what you get in Delaware County. Everybody gets each other's back here," said John Port of The Delco Group. What you can do The community has also raised tens of thousands of dollars in a gofundme campaign which will now help support KJ's medical needs moving forward.
Indian Express
27-05-2025
- Health
- Indian Express
Explained: A first— how a customised gene-editing tool was used to treat 9-month-old boy
A nine-month-old boy, born with a rare genetic disorder, has become the first (known) person to successfully receive a custom gene-editing treatment, a report published on May 15 in the New England Journal of Medicine said. Kyle 'KJ' Muldoon Jr suffers from CPS1 deficiency which causes toxic levels of ammonia to accumulate in his blood. To treat him, scientists and doctors from the University of Pennsylvania and the Children's Hospital of Philadelphia developed a personalised treatment based on 'base editing', a new version of the decade-old CRISPR-Cas9 technology. Scientists say this technology can potentially treat thousands of uncommon genetic diseases. But there remain many roadblocks to its universal adoption. What is CRISPR? Following infection by a virus, humans generate an 'immune memory' in the form of antibodies. When they are infected by the same virus again, these antibodies quickly identify the pathogens and help neutralise them. CRISPR, short for 'clustered regularly interspaced short palindromic repeats', is an immune system found in microbes such as bacteria which fights invading viruses. When a virus infects a bacterial cell, CRISPR too helps establish a memory — but a genetic one, not in the form of antibodies like in humans. When a virus enters a bacterial cell, the bacterium takes a piece of the virus's genome and inserts the DNA into its own genome. CRISPR then produces a new 'guide' RNA with the help of the newly acquired DNA. During a future attack by the same virus, the guide RNA quickly recognises the virus DNA and attaches itself to it. Then, the guide RNA directs an enzyme (a type of protein) called Cas9 to act like 'molecular scissors' to cut and eliminate the virus DNA. In 2012, scientists Jennifer Doudna and Emmanuelle Charpentier replicated this mechanism found in microbes to develop a gene-editing tool, which they called CRISPR-Cas9, a feat which earned them the Nobel Prize for Chemistry eight years later. How does CRISPR-Cas9 gene-editing work? The tool works much like the 'cut-copy-paste', or 'find-replace' functionalities in common computer programmes. Genetic information in DNA is stored as code made up of four chemical bases — adenine (A), guanine (G), cytosine (C), and thymine (T). These bases exist in pairs, which are then stacked one on top of each other, creating the horizontal layers of the double-helix structure of DNA. Note that A always pairs with T, and C always pairs with G. Genetic disorders, like the one KJ suffers from, occur due to the presence of an abnormal DNA sequence, that is, a mispairing (A-G or G-T). The first task for the gene-editing tool is to identify the abnormal DNA sequence behind a patient's ailment. Once the bad DNA is located, scientists create a guide RNA attached to a Cas9 enzyme, which is then introduced to the target cells of the patient. The guide RNA recognises the bad DNA sequence, then the Cas9 enzyme cuts the DNA at the specified location in a process called a 'double-strand break' (since the cut is made on both strands of the DNA). This gets rid of the DNA sequence causing the illness. DNA strands have a natural tendency to reattach and repair themselves, meaning there is a chance that the bad sequence regrows. To tackle this issue, scientists also supply the correct DNA sequence after the 'cutting' process which is meant to attach itself to the broken strands of DNA. Over the years, scientists have made many improvements to the original CRISPR-Cas9 technology, making it safer and more precise. A newer, evolved version of this tool is 'base editing'. How does base editing work? Base editing and CRISPR-Cas9 differ significantly in how they modify DNA. Unlike CRISPR-Cas9, base editing does not make a double-strand break. Rather, it enables targeted single-base conversions with the help of a Cas9 enzyme fused to a base-modifying enzyme. This allows scientists to fix mispairing of the bases by changing one specific base. For instance, mispaired A-C bases can be corrected to A-T by converting C to T. To treat KJ, scientists first determined which mispaired base in his DNA was causing his condition. They then programmed the base editing tool to find and rewrite the target base. This process can be likened to using a pencil and an eraser, rather than scissors and glue, as in CRISPR-Cas9. 'In the older version of CRISPR, scientists were required to provide additional DNA from outside, which would be pasted at the site where the double-strand break takes place. In base editing, however, the system by itself can make a very precise change without the need for a foreign DNA to be inserted,' Debojyoti Chakraborty, principal scientist at CSIR-Institute of Genomics and Integrative Biology, told The Indian Express. 'As a result, base editing has fewer components and is compact, making it easier to package in delivery vehicles, which can take it to target cells,' he said. In 2023, Chakraborty and his team tried to develop a similar tool to treat a patient with a rare neurodegenerative disease. But she passed away before the experiment could be carried out. Will base editing become commonplace soon? Chakraborty said the successful use of base editing for treating KJ has given hope to doctors treating people with rare genetic disorders for whom no medical treatments were currently available. However, it is unlikely that such technologies will become commonplace any time soon, first and foremost due to the prohibitive costs of such treatments. Even if it were to become widely available, base editing would not be accessible to most people. (KJ's treatment was funded by research institutes and biotechnology. While they did not make any official disclosure regarding its cost, it is likely to be in the range of hundreds of thousand dollars, maybe more). Also, the base editing tool created to help KJ was a one-off treatment, meaning it was designed specifically for his unique genetic disorder and cannot be used to treat other individuals with different disorders. This poses a unique challenge with regards to scaling up such technologies for mass consumption, something that disincentives pharmaceutical companies to invest in their development. Getting regulatory approvals is another issue. 'To do such a thing in India is very difficult because it also means that you will have to get rid of red tapism,' Chakraborty said. It remains to be seen how researchers make such personalised treatments more accessible. Till then, only a few fortunate people like KJ will benefit from base editing therapies.
Gulf Today
21-05-2025
- Health
- Gulf Today
US baby with rare illness treated with tailor-made gene edit
A US infant with a rare condition has become history's first patient to be treated with a personalized gene-editing technique that raises hopes for other people with obscure illnesses, doctors said Thursday. The wee pioneer is KJ Muldoon, now a 9-and-a-half-month-old boy with chubby cheeks and big blue eyes. Shortly after birth, he was diagnosed with a rare and serious condition called CPS1 deficiency. It is caused by a mutation in a gene that produces an enzyme key to liver function, and prevents people with it from eliminating certain kinds of toxic waste produced by their metabolism. "You Google 'CPS1 deficiency' and it's either fatality rate or liver transplant," the baby's mother, Nicole Muldoon, says in a video released by Children's Hospital of Philadelphia, where the baby was treated. KJ Muldoon (centre) sits with his siblings. AP With the prognosis grim, doctors suggested something that had never been done before: a personalized treatment to fix the baby's genome using what amounts to a pair of molecular scissors -- the technique called Crispr-Cas9, which earned its creators the Nobel prize for chemistry in 2020. The boy's father said he and his wife faced an impossible decision. "Our child is sick. We either have to get a liver transplant or give him this medicine that's never been given to anybody before, right?" said Kyle Muldoon. In the end, they agreed to have the child treated with an infusion created just for him to fix his genetic mutation -- incorrect DNA letters in the several billion that make up the human genome. "The drug is really designed only for KJ, so the genetic variants that he has are specific to him. It's personalized medicine," said Rebecca Ahrens-Nicklas, a member of the medical team who specializes in pediatric genetics. KJ Muldoon sits with his parents, Kyle and Nicole Muldoon, and his siblings. AP Once the tailor-made infusion reaches the liver, the molecular scissors contained in it penetrates cells and goes to work editing the boy's flawed gene. The results were promising for other people with genetic conditions, said the medical team, which published their study Thursday in the New England Journal of Medicine. KJ can now follow a diet richer in proteins -- his condition prohibited such before -- and does not need as much medicine as he used to. But he will need to follow-up long term to monitor the safety and efficacy of the treatment, the team said. Ahrens-Nicklas said she hoped this achievement will allow the boy to get by with little or no medication some day. "We hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient's needs," the doctor said. Agence France-Presse
The Print
20-05-2025
- Health
- The Print
Much before US baby, Gurugram girl could've been 1st to get personalised gene editing. But time ran out
Rajeev, founder of a software company, also said it was 'painful' to have come across the news about the medical milestone that benefitted a rare disease patient in the US. 'My daughter missed a similar breakthrough by a whisker,' he rued. Their daughter, Uditi, suffering from another devastating rare genetic disorder, could have been the world's first patient to receive a similarly personalised treatment—and, hopefully, still alive—had time not run out. 'I wish Uditi were born a few years later or this treatment would have been successful a little earlier,' Sonam said. 'We had reached so close, but in the end it turned out to be quite far.' New Delhi: When Gurugram residents Sonam and Rajeev Saraf first read the news about scientists and doctors in the US rewriting a baby's faulty DNA, using the world's first customised gene-editing therapy, to treat a rare genetic disorder three days ago, they cried. 'I could not finish reading it. I broke down,' Sonam told ThePrint. Specialists at the Children's Hospital of Philadelphia and the University of Pennsylvania in the US became the first globally to treat a baby named KJ with a customised gene-editing therapy, after diagnosing the child with a severe genetic disorder that kills about half of those affected in early infancy. The therapy was administered using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, also referred to as 'genetic scissors' that won its inventors the Nobel Prize in 2020. KJ's disorder, known as CPS1 deficiency, is caused by the lack of an enzyme that leads to dangerous build-up of ammonia because of the natural breakdown of protein from food, and can cause severe damage to the liver and brain, among other vital organs. The latest breakthrough involved scientists identifying specific mutations causing the disease, designing a gene editing therapy to correct them, and testing the treatment and nanoparticles specially created to deliver the treatment to the liver. The procedure, named base editing, allows the alteration of DNA or molecular instructions. Their ground-breaking work was published in The New England Journal of Medicine on 15 May. The feat is being celebrated worldwide as it demonstrates the treatment's potential of fixing many life-threatening genetic diseases by rewriting faulty DNA. 'Some years ago, all this would have been in the realm of science fiction, but today it is reality and I am very happy,' molecular biologist Dr Debjyoti Chakraborty, who is running multiple research projects using CRISPR technology in India, told ThePrint. Associated with the New Delhi-based Centre for Scientific and Industrial Research in India-Institute of Genomics and Integrative Biology (CSIR-IGIB), Chakraborty was leading a project to develop a similar treatment for Uditi from 2022. 'This news, however, has also brought a feeling of disappointment as time ran out in our case,' said the scientist. In 2023, when Uditi passed away, scientists leading India's two leading gene therapy laboratories—one at CSIR-IGIB and the other at Narayana Nethralaya in Bengaluru—were nearly ready with an experimental treatment using base gene editing specifically designed for Uditi. Also Read: Scientists find naturally existing DNA editing tool in all life, say it increases scope beyond CRISPR A race against time The Sarafs were funding the research to develop the world's first personalised treatment in India, using cutting-edge therapy, in a desperate bid to save their daughter. Uditi was suffering from an extremely rare neurogenetic condition called FENIB or familial encephalopathy with neuroserpin inclusion bodies. She was 20 when she passed away in October 2023. FENIB is a fatal neurodegenerative disease primarily caused by the aggregation of mutant neuroserpin proteins, which disrupt the functions of neurons within brain cells, leading to progressive dementia, seizures and, eventually, death. This condition usually manifests in those affected between the second to fifth decades of life, and in some, the progression is rapid. It is mostly misdiagnosed due to difficulties in connecting it to the genetic root. There is no treatment available to treat the condition. Uditi, a normal child until nine, was first diagnosed as an epilepsy patient in 2011. Investigations over several years later confirmed she had the rare condition due to a faulty gene. The Sarafs, who were witnessing Uditi's gradual deterioration, first took her to the US and reached out to top genomic scientists to get a treatment tailor-made for her. 'She was a bright child early on, but as her condition progressed, she started lagging behind in studies. We first enrolled her in a special school in Gurugram but later decided to take her to the US for both treatment and education,' Sonam said. The couple approached top genomic scientists and epilepsy specialists in the US after coming across news about the rapid advances in gene therapy. The work to treat Uditi's condition started in 2017, when a globally renowned epilepsy specialist and researcher at New York University (NYU), Orrin Devinsky, told the Sarafs that in theory it was possible to correct the mutation leading to Uditi's disease using base editing, which had not yet been tested in a clinical trial. There were, however, challenges in implementing it, including securing a funding grant and ensuring the successful delivery to brain cells. NYU researchers with expertise in genome editing, however, finally undertook the project with funding being pulled out from some other projects at the centre, as well as partial support from the Sarafs. And work began to genetically engineer Uditi's FENIB mutation into cells grown in the lab, the first part of the complex procedure. In late 2019, the family decided to move back to India when they found staying in the US too expensive, the research work was not as fast as expected and because of Uditi's growing desire to be closer to the extended family. Uditi's condition showed no improvement and a month in the Intensive Care Unit (ICU) at a Gurugram hospital following a COVID-19 infection in 2021, where she was isolated and cut off from her family, led to a rapid decline from which there was little chance of coming back. A little later, Sonam reached out to Chakraborty's lab at CSIR-IGIB after coming across news that it was working on developing treatment for some conditions using CRISPR technology. The lab was ready to take up the research work but funding was a big hurdle. The Sarafs offered to pitch in, pledging about Rs 4.5 crore, the estimated amount needed for developing the treatment before the actual trials. The project saw the coming together of gene editing labs at CSIR-IGIB and Narayan Nethralaya. 'We had tested the therapy in animal models and were in the process of seeking regulatory approval for testing the experimental therapy in Uditi when we lost her,' Dr Arkasubhra Ghosh, chief scientist, molecular signalling and gene therapy, Grow Research Laboratory, Narayana Nethryalaya in Bengaluru, told ThePrint. Parallelly, talks were also on to put together a team of neurosurgeons who would have administered the therapy to Uditi. 'We were able to carry out work in one-and-a-half years that would have normally taken three to five years, just to be able to save Uditi, whose condition was fast deteriorating, he added. Tragic end of a scientific sprint Uditi's condition was caused by mutations in the SERPINI1 gene, which provides instructions for making a protein called neuroserpin found in nerve cells, explained genomic scientist Sridhar Sivasubbu. He is associated with the Indian Institute of Technology (IIT) Kanpur and the Vishwanath Cancer Care Foundation. People with FENIB produce an abnormal and unstable form of neuroserpin that clumps together within neurons, forming what are known as Collins bodies or inclusion bodies. These clumps disrupt neuronal function and gradually lead to cell death. As more and more neurons die, cognitive abilities as well as the normal functioning of a patient decline. Till her 19th birthday, Uditi was largely up and about, though she had lost most of her mental abilities. But by the time she turned 20, she was bedridden and barely conscious. The collaboration to find a treatment for her saw Chakraborty's lab working on editing enzymes that could fix the mutation in the SERPINI1 gene using a low-cost CRISPR technology system to deliver the editing system into the cells. Ghosh's lab, which had developed and patented a recombinant adeno-associated viral vector or AVV system to deliver the edited enzymes into the body, was another important arm of the project. As part of the project, scientists generated stem cells from samples of Uditi's blood, which were then converted to neurons. They then used base editing on them in the lab. Ghosh's lab worked on readying the AAV that could be used to transport the CRISPR components into Uditi's neurons. It was painstaking work. Researchers faced challenges in determining which strain of AAV would work better and be safer. For this, several types of AAVs were tested in mice. Eventually, a type of vector (AAV 9) was selected and was ready to be injected into Uditi's brain. But before that, Uditi developed aspiration pneumonia, a type of pneumonia caused by inhaling food, liquid, saliva, or stomach contents into the lungs, leading to infection. She died within a few weeks of developing the condition. It is common in those with swallowing difficulties, poor gag reflex, or other medical conditions, symptoms common in those in advanced stages of severe FENIB. This brought the scientific projects led by Chakraborty and Ghosh to an abrupt pause. Their work, however, based on successful results in animal models, is currently under review for publication in a leading international medical journal. 'I still hope that the treatment that was being devised for Uditi helps other FENIB patients and our support to scientists working on the project continues,' Sonam said. It was painstaking work. Researchers faced challenges in determining which strain of AAV would work better and be safer. For this, several types of AAVs were tested in mice. Eventually, a type of vector (AAV 9) was selected and was ready to be injected into Uditi's brain. Dr Souvik Maiti, a noted nucleic acid biophysicist and a co-developer of the CRISPR protein that was being used for Uditi ensured that no bottlenecks in scientific, administrative and funding related issues would arise by streamlining all resources within and outside the institute. Maiti, as of now, is the director at CSIR-IGIB. CRISPR: a hope for the future Explaining KJ's condition, scientists say that during the normal breakdown of proteins in the body, ammonia is naturally produced. Normally, our bodies know how to convert the ammonia to urea and then excrete that urea through urination. But a child with a urea cycle disorder lacks an enzyme in the liver needed to convert ammonia to urea. After years of preclinical research with similar disease-causing variants, specialists targeted KJ's specific variant of CPS1, which was recognised soon after his birth. Within six months, their team designed and manufactured a base editing therapy delivered via lipid nanoparticles to the liver to correct the sick child's faulty enzyme. In late February this year, KJ received his first infusion of this experimental therapy, and since then, he has received follow-up doses in March and April 2025. In the short time since treatment, according to publicly available information, KJ has shown to tolerate increased dietary protein and needs less nitrogen scavenger medication. Yet, doubts remain as this is only an experimental treatment. 'I feel the need to be conservative about how this success should be perceived, especially since most patients will never be able to get such a therapy in their lifetimes due to the complications surrounding affordability and accessibility of this therapy,' Chakraborty said. The scientist added that only time will tell if the treatment will translate to a life free from the disease. In December 2023, the US Food and Drug Administration approved Casgevy by Vertex Pharmaceuticals and Lyfgenia by Bluebird Bio—the first gene therapies for sickle cell disease (SCD) in patients 12 years and older. This marked a milestone in medical advancement in treating a debilitating disease, primarily affecting the capacity of red blood cells to carry adequate oxygen across the body, with the use of innovative cell-based gene therapies. But Chakraborty said affordability remains a challenge worldwide, with the cost of treatment estimated at around $2-3 million per patient (Rs 17-25 crore). For the scientist, who has been working to develop a treatment for SCD and some other rare conditions using CRISPR, the challenge is to develop it at a fraction of this cost. 'This promising medical technology will not be of any use in a country like ours if we cannot make it affordable,' he said. (Edited by Sugita Katyal) Also Read: Gene-editing cures progeria in mice — a fatal condition Amitabh Bachchan portrayed in 'Paa'
Japan Today
18-05-2025
- Health
- Japan Today
Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions
This photo provided by the Children's Hospital of Philadelphia shows KJ Muldoon after a follow up dose of an experimental gene editing treatment at the hospital in April 2025. (Chloe Dawson/Children's Hospital of Philadelphia via AP) By LAURA UNGAR A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, saying he's among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his genetic code that kills half of affected infants. Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can someday help the millions left behind even as genetic medicine has advanced because their conditions are so rare. 'This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments,' said Dr. Kiran Musunuru, a University of Pennsylvania gene editing expert who co-authored the study published Thursday in the New England Journal of Medicine. The baby, KJ Muldoon of Clifton Heights, Pennsylvania, is one of 350 million people worldwide with rare diseases, most of which are genetic. He was diagnosed shortly after birth with severe CPS1 deficiency, estimated by some experts to affect around one in a million babies. Those infants lack an enzyme needed to help remove ammonia from the body, so it can build up in their blood and become toxic. A liver transplant is an option for some. Knowing KJ's odds, parents Kyle and Nicole Muldoon, both 34, worried they could lose him. 'We were, like, you know, weighing all the options, asking all the questions for either the liver transplant, which is invasive, or something that's never been done before,' Nicole said. 'We prayed, we talked to people, we gathered information, and we eventually decided that this was the way we were going to go,' her husband added. Within six months, the team at Children's Hospital of Philadelphia and Penn Medicine, along with their partners, created a therapy designed to correct KJ's faulty gene. They used CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020. Instead of cutting the DNA strand like the first CRISPR approaches, doctors employed a technique that flips the mutated DNA 'letter' — also known as a base — to the correct type. Known as 'base editing," it reduces the risk of unintended genetic changes. It's 'very exciting' that the team created the therapy so quickly, said gene therapy researcher Senthil Bhoopalan at St. Jude Children's Research Hospital in Memphis, who wasn't involved in the study. 'This really sets the pace and the benchmark for such approaches.' In February, KJ got his first IV infusion with the gene editing therapy, delivered through tiny fatty droplets called lipid nanoparticles that are taken up by liver cells. While the room was abuzz with excitement that day, 'he slept through the entire thing,' recalled study author Dr. Rebecca Ahrens-Nicklas, a gene therapy expert at CHOP. After follow-up doses in March and April, KJ has been able to eat more normally and has recovered well from illnesses like colds, which can strain the body and exacerbate symptoms of CPS1. The 9 ½-month old also takes less medication. Considering his poor prognosis earlier, 'any time we see even the smallest milestone that he's meeting – like a little wave or rolling over – that's a big moment for us,' his mother said. Still, researchers caution that it's only been a few months. They'll need to watch him for years. 'We're still very much in the early stages of understanding what this medication may have done for KJ,' Ahrens-Nicklas said. 'But every day, he's showing us signs that he's growing and thriving.' Researchers hope what they learn from KJ will help other rare disease patients. Gene therapies, which can be extremely expensive to develop, generally target more common disorders in part for simple financial reasons: more patients mean potentially more sales, which can help pay the development costs and generate more profit. The first CRISPR therapy approved by the U.S. Food and Drug Administration, for example, treats sickle cell disease, a painful blood disorder affecting millions worldwide. Musunuru said his team's work — funded in part by the National Institutes of Health — showed that creating a custom treatment doesn't have to be prohibitively expensive. The cost was 'not far off' from the $800,000-plus for an average liver transplant and related care, he said. 'As we get better and better at making these therapies and shorten the time frame even more, economies of scale will kick in and I would expect the costs to come down,' Musunuru said. Scientists also won't have to redo all the initial work every time they create a customized therapy, Bhoopalan said, so this research 'sets the stage' for treating other rare conditions. Carlos Moraes, a neurology professor at the University of Miami who wasn't involved with the study, said research like this opens the door to more advances. 'Once someone comes with a breakthrough like this, it will take no time" for other teams to apply the lessons and move forward, he said. 'There are barriers, but I predict that they are going to be crossed in the next five to 10 years. Then the whole field will move as a block because we're pretty much ready.' © Copyright 2025 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed without permission.
