“It’s a very different way of altering genomes that is controllable. The team of doctors and nurses caring for him were stunned by his rapid decline and death. 1. When he arrived in the surgical intensive care unit at 8 Wednesday morning, Raper and Batshaw told him that dialysis had brought Jesse's ammonia level down to 72 but that other complications were developing. Paul Gelsinger planned to fly in a week later for the liver biopsy, which he considered the trial's most serious risk. Caplan says parents of dying infants are incapable of giving informed consent: ''They are coerced by the disease of their child.'' ''We had got ourselves all hyped up,'' Anderson now admits, ''thinking there would be rapid, quick, easy, early cures. The jagged peak of Mount Wrightson towers 9,450 feet above Tucson, overlooking a deep gorge where the prickly pear cactus that dots the desert floor gives way to a lush forest of ponderosa pine. He took one duffel bag full of clothes and another full of wrestling videos. To begin thinking about some of the ethical issues in gene therapy research, and human experimentation in general, we explore the following real-life case. Many patients with devastating diseases, such as muscular dystrophy, cystic fibrosis, and Huntington’s disease, as well as certain cancers and rare diseases for which few treatments are available, will accept the unknown chance they’ll experience some harm from an experimental therapy if it also might lessen their symptoms or extend their lives. By the time Batshaw joined the faculty at Penn in 1988, he was dreaming of a cure -- gene therapy. So we don’t even bring those forward.”. Jesse Gelsinger’s father, Paul, testifies at a Senate subcommittee hearing on gene therapy and patient risk, February 2000. Stolberg SG. Within a day he became disoriented and showed signs of jaundice. Jesse was the most public face of the group and he started to get cocky. Batshaw was banging on Wilson's door even before Wilson arrived at Penn in March 1993, and within a month they were collaborating on studies of OTC-deficient mice. To Wilson, the answer seemed obvious: sick babies. In 1990 Owens was posthumously awarded the Congressional Gold Medal. The news that an experimental treatment had killed a basically healthy volunteer rocked the field of gene therapy and the broader world of biological research. On September 13, 1999, Jesse was infused with the corrective OTC into his hepatic artery. “That made the whole field of gene therapy go away, mostly, for at least a decade. The room was crowded with equipment and people: 7 of Paul's 15 siblings came in, plus an array of doctors and nurses. He suffered from ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder, but it was controlled with a low-protein diet and drugs, 32 pills a day. He left his wife a note and walked the half mile to the Penn medical center to see Jesse. already reviewed gene-therapy proposals. Then he put on scrubs, gloves and a mask and went in to see his son. Researchers at the University of Pennsylvania in Philadelphia were developing a fix for the OTC gene, which produces an enzyme that prevents ammonia buildup. Background. '', Of the three, Batshaw seems to have taken it the hardest. News coverage portrayed the trial researchers as overeager and undercautious, taking shortcuts and disregarding rules meant to protect the people in their care. ''That,'' Raper says, ''would be the best tribute to Jesse. The package may include other components, such as a new piece of DNA code to plug into the edited area. Maybe they'll come up with a cure.'''. There are some circumstances where in our attempt to make them more efficient, their safety profile was compromised. The experiment stood in stark contrast to others that had earned Varmus's scorn. Jesse Gelsinger was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was two years old. And while there is no simple answer, there is also no shortage of possible candidates: the investigators at the University of Pennsylvania who carried out the experimental therapy; the local institutional review board that purportedly omitted animal data in the informed … “Some would call it kind of irrational exuberance,” he said in his interview with Distillations. Now gene therapy has Jesse Gelsinger. A clinical trial of a CRISPR-based treatment for color blindness, for example, might not be worth the risk. Raper was beside himself. The doctors are still investigating; their current hypothesis is that the adenovirus triggered an overwhelming inflammatory reaction -- in essence, an immune-system revolt. No one, perhaps, is more acutely aware of gene therapy's broken promise than Mark Batshaw, the pediatrician who proposed the experiment that cost Jesse Gelsinger his life. ''It was not something we had seen before,'' Raper says. Ten years ago, Jesse Gelsinger died while participating in a human gene therapy trial at the University of Pennsylvania (“Penn”). But one day last December, Paul Gelsinger arrived home to find his son curled up on the couch. As far as government officials know, Jesse's death on Sept. 17 was the first directly related to gene therapy. '', The Batshaw-Wilson protocol was among the last the committee would ever approve. On the morning of Friday the 17th, a test showed that Jesse was brain dead. / Wilson, Robin Fretwell . That night, Jesse was sick to his stomach and spiked a fever, 104.5 degrees. September 17 marked 20 years since the death of 19-year-old Jesse Gelsinger in a gene therapy trial. His death came to signify the corrosive influence of financial interests in human subjects research. director, who won the Nobel Prize for his discovery of a family of cancer-causing genes, had made no secret of his distaste for the conduct of gene-therapy researchers. Researchers hadn’t told Jesse about the earlier patients’ side effects or about two lab monkeys killed by high doses of adenoviruses. It had a ''ZIP code,'' on it, Batshaw says, that would carry it straight to the liver. A few of Gelsinger’s enzymes had the ability to function normally, whereas other cells had an OTC gene with a large deletion. '', See the article in its original context from. '', Wilson reported the death immediately, drawing praise from government officials but criticism from Arthur Caplan, who says they should have made the news public, in a news conference. View Homework Help - HW+5+solutions from CENG 1600 at The Hong Kong University of Science and Technology. But when the team contemplated testing in people, they ran smack into an ethical quandary: who should be their subjects? We’ve made a lot of advances in making them better. Those discoveries have been crucial to the production of a new generation of medicines. Patients were dreaming, too, says Tish Simon, former co-president of the National Urea Cycle Disorders Foundation, whose son died of OTC deficiency three years ago. Half of them die within a month. When considering a middle name, we pondered James, after his grandfather, but decided that just Jesse was enough for this kid. At half past noon, he was done. Paul Gelsinger called; he and Jesse talked briefly, exchanging I love yous. Still, it offered hope, the promise that someday Jesse might be rid of the cumbersome medications and diet so restrictive that half a hot dog was a treat. He knew when he signed up for the experiment at the University of Pennsylvania that he would not benefit; the study was to test the safety of a treatment for babies with a fatal form of his disorder. Jesse Gelsinger did not inherit the disease; he was diagnosed with OTCD as a young child. That doesn’t mean clinical trials of CRISPR-based therapies shouldn’t happen, but it does affect the risk-benefit calculation, he said. For example, Editas Medicine, a gene-therapy company based in Cambridge, Massachusetts, uses one of Wilson’s vectors to deliver its treatment for Leber’s congenital amaurosis type 10, an inherited blindness condition. He had an intense inflammatory response and developed a dangerous blood-clotting disorder, followed by kidney, liver, and lung failure. That question cannot be answered until all the data are analyzed. Gelsinger may refer to: . The initial goal was simple: to cure, or prevent, these illnesses by replacing defective genes with healthy ones. Patients would be injected with working copies of the gene that had been attached to an adenovirus, a type of cold virus. Raper was not particularly surprised: other patients had experienced the same reaction. As of August, the government had reviewed 331 gene-therapy protocols involving more than 4,000 patients. So when a doctor told Jesse that a clinical trial for a potential OTCD treatment was in the works, he was very interested. AIDS had Magic Johnson. But how safe is safe enough? But perhaps most damning were failures in the informed-consent process. As a graduate student in biological chemistry, Wilson had taken a keen interest in rare genetic diseases. Paul Gelsinger didn't need to be told: ''I knew it already.'' That decision, however, was later reversed by the F.D.A., which insisted that because the adenovirus would travel through the blood and wind up in the liver anyway, the original plan was safer. Eighteen-year-old Jesse Gelsinger, three months before his death. This condition is known as mosaicism. There are a number of possible explanations, he says: the vector may have reacted badly with Jesse's medication; Jesse's status as a mosaic may have played a role; or perhaps the early testing in monkeys, which showed that the stronger vector had deleterious side effects, was more of a harbinger of danger than the doctors realized. Since then, there have been some accomplishments: a team at Tufts University has used gene therapy to grow new blood vessels for heart disease patients, for instance. Both Erickson and the other scientific reviewer thought the experiment was too risky to test on asymptomatic volunteers and recommended rejection. The exact cause of his death was from adult respiratory … You just can’t know. Using that particular virus helps ensure the therapy only penetrates (is trophic for) the vision cells that need to be altered and doesn’t end up causing effects elsewhere. The treatments worked, but within a few years four of the children developed leukemia and one of them died. Biochemist Jennifer Doudna, who later discovered the CRISPR-Cas9 gene-editing mechanism, remembers feeling the shock waves as a young researcher, even though her work had nothing to do with gene therapy or any kind of medical research. How did the death of the Jesse Gelsinger affect the progress of gene What you don’t see are all the failed experiments. '', Among those keeping a close eye on Anderson's debut was Jim Wilson, a square-jawed, sandy-haired Midwesterner who decided to follow his father's footsteps in medicine when he realized he wasn't going to make it in football. The Gelsinger Case. ''I will look to you here often, Jess,'' Paul Gelsinger said sadly. It had been tried on only 1,000 people before, Raper says. But the researchers’ experimental treatment had lengthened the lives of lab mice bred to be deficient in OTC enzymes, and the scientists were hopeful the gene-repair method they were testing could eventually be used to treat many liver diseases. “I really thought a lot about, should we try to make the more efficient vectors safer, or should we make the safer vectors more efficient?” he said in a phone interview. Batshaw was a young postdoctoral fellow when he met his first urea-cycle-disorder patient in 1973, correctly diagnosing the disease at a time when most other doctors had never heard of it. He suffered from ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder, but it was controlled with a low-protein diet and drugs, 32 pills a day. Last year, for example, two groups of researchers said they found a possible problem when they tested CRISPR on retinal cells and stem cells. Still, it made Erickson, one of two scientists assigned by the RAC to review the experiment, uneasy. Adenovirus was the right size: when its viral genes were excised, the OTC gene fit right in. Every realm of medicine has its defining moment, often with a human face attached. The tragedy sent shock waves through Philadelphia’s biotech industry and across the medial world. Gelsinger obtained OTCD, apparently as the result of a spontaneous genetic mutation after conception. February 2, 2000 | Clip Of Safety of Gene Therapy This clip, title, and description were not created by C-SPAN. ''I think it's a perilous time for gene therapy,'' says LeRoy Walters, a bioethicist at Georgetown University and former chairman of the RAC. The urea cycle is a series of five liver enzymes that help rid the body of ammonia, a toxic breakdown product of protein. Four days after receiving the shot Jesse was declared brain dead and taken off life support. But he now balances such celebration with hard-earned prudence. So it was here, on a clear Sunday afternoon in early November, that Paul Gelsinger laid his 18-year-old son to rest, seven weeks after a gene-therapy experiment cost him his life. When Raper got to the hospital, about 6:15 a.m., he noticed that the whites of Jesse's eyes were yellow. The ceremony was simple and impromptu. The university also paid the federal government a $514,000 settlement. Those were the last words they ever spoke. Wilson said the question of whether a gene edit could inadvertently cause mutations elsewhere in the chromosome and cause cancer in a patient, much as SCID gene therapy caused leukemia, will not be resolved soon. TimesMachine is an exclusive benefit for home delivery and digital subscribers. It turned out Jesse’s pretrial test results showed he had poor liver function, indicating he arguably shouldn’t have received the OTC gene injection. In 2018 Wilson published a paper warning that animals receiving high doses of AAVs in tests of a therapy for spinal muscular atrophy sustained nerve and liver damage, raising questions about the volume of viruses humans can safely handle. Kaabali received Luxturna, which treats a form of hereditary blindness. The mouse experiments were encouraging. Transplant surgery had Barney Clark, the Seattle dentist with the artificial heart. Wilson was also accused of a conflict of interest: he had a stake in the company that owned the gene-transfer technology and stood to benefit if the trial succeeded. The ECMO, Raper reported, appeared to be working. In the weeks after Jesse’s death James Wilson, the director of the University of Pennsylvania’s Institute for Human Gene Therapy, and the other doctors involved in the trial tried to understand what happened. In a flash the field of gene therapy collapsed, taking its grandiose promises of miracle cures along with it. Wilson's biggest fear was that Jesse died as a result of human error, but so far there has been no evidence of that. '', Gene therapy became a reality on Sept. 14, 1990, in a hospital room at the National Institutes of Health, in Bethesda, Md., when a 4-year-old girl with a severe immune-system deficiency received a 30-minute infusion of white blood cells that had been engineered to contain copies of the gene she lacked. That night, at his hotel, Paul Gelsinger couldn't sleep. It was a striking reversal for a renowned scientific pioneer. Ten years ago, Jesse Gelsinger died while participating in a human gene therapy trial at the University of Pennsylvania (“Penn”). In other words, CRISPR apparently subverted one of the body’s disease-fighting mechanisms, making healthy cells die and allowing potentially cancerous ones to remain. A trim 47-year-old with intense blue eyes, Gelsinger, who makes his living as a handyman, gained custody of his four children nine years ago, when he divorced their mother, who suffers from manic depression. Most suffer severe brain damage. Until Jesse died, gene therapy was a promising idea that had so far failed to deliver. He experienced a severe immune reaction to the vector and died four days after the infusion (Sibbald, 2001). How can anybody, he thought, survive this? The goal was to find what Wilson calls ''the maximum tolerated dose,'' one high enough to get the gene to work, but low enough to spare patients serious side effects. That’s going to be a huge challenge,” he said. Born on June 18, 1981, Jesse Gelsinger was a real character in a lot of ways. “We cannot say there’s zero exposure to the rest of the body,” Editas CEO Katrine Bosley said during a talk at the Science History Institute last fall. '', Paul Gelsinger does not hold the doctors responsible, although he is acutely interested in knowing what other scientists knew about adenovirus before Jesse died. Jesse Gelsinger was not sick before died. They drew her white blood cells, used a retrovirus to insert a working gene into the cells, then injected them back into her body, which helped give her a functioning immune system. The virus, altered to be harmless, would infect the patients’ liver cells and integrate the added gene into their chromosomal DNA. ''I die, and it's for the babies.''. Rarely in modern medicine has an experiment been filled with so much hope; news of the treatment ricocheted off front pages around the world. “We were all very much aware of what happened there and what a tragedy that was,” she said in a recent interview. ''If they made a mistake, you would feel a little safer. The drug, Zolgensma, treats spinal muscular atrophy, an inherited disease that destroys nerve cells and is the most common genetic cause of death of infants. Arthur Caplan, the university's resident bioethics expert, thought otherwise. Jesse Gelsinger was not sick before died. Jesse Gelsinger (18. kesäkuuta 1981 – 17. syyskuuta 1999) oli ensimmäinen geeniterapiaan kuollut ihminen. true. They wanted to paralyze his muscles and induce a deeper coma, so that a ventilator could breathe for him. It was aimed at a rare genetic disease, not cancer or AIDS. He wanted to sign up right away. Jesse was the kind of kid who kept $10.10 in his bank account -- You need $10 to keep it open,'' Gelsinger explained -- but those assembled on the mountaintop agreed that he had a sharp wit and a sensitive heart. As exciting as it is, I really would like to see . . . Wilson briefly considered leaving science entirely. He speaks frequently of God, and of ''purity of intent,'' which is his way of saying that Jesse demonstrated an altruism the rest of us might do well to emulate. An early dietitian set out to prove that vegetarian cooking was good for the body. Enrollment in those trials was suspended by the Food and Drug Administration after Jesse's death. Those that lacked the treatment died. Batshaw went back to Washington. 1999 Nov 28:136-140, 149-150. But how safe is safe enough for the patients testing these therapies? Babies born with OTCD usually fall into comas soon after birth and suffer brain damage. And they outlined the major risks: bleeding, from either the gene-therapy site or a subsequent liver biopsy, which would require surgery; or serious liver inflammation, which could require an organ transplant and might lead to death. Adenovirus seemed a logical choice. As a child, Batshaw struggled with hyperactivity: he didn't read until the third grade; in the fourth, his teacher grew so irritated at his constant chatter that she stuck his chair out in the hall. “Of course, the challenge is that patients are waiting, so you don’t want to delay unduly. The retroviral vectors had been integrated near oncogenes, which can cause cancer, apparently triggering the leukemia. Ten years ago, Jesse Gelsinger died while participating in a human gene- therapy trial at the University of Pennsylvania ("Penn"). Jesse Gelsinger (1981–1999), American, the first person publicly identified as having died in a clinical trial for gene therapy; Pat Gelsinger, author, CEO of VMware He had been vomiting uncontrollably, a sign, Paul knew, that Jesse's ammonia was rising. That ill-fated rush to experiment on human subjects was driven by simplistic modeling suggesting the approach “ought to” work, as well as the “fervent hopes” of charitable foundations seeking cures for lethal diseases, he wrote. The biotech death of Jesse Gelsinger. ''That's what's so frightening,'' French Anderson says. Me. They played tourists, visiting the Liberty Bell and the Rocky statue, where Jesse was photographed, fists raised, a picture that would circulate in the newspapers after his death. ''We felt pretty compelled by that.'' At 54, Batshaw, who left the University of Pennsylvania last year for Children's National Medical Center, in Washington, is tall and gangly with slightly stooped shoulders and a shy smile that gives him the air of an awkward schoolboy, which he once was. Headquartered in a century-old building amid the leafy maple trees and brick sidewalks of the picturesque Penn campus, the six-year-old institute, with 250 employees, state-of-the-art laboratories and a $25 million annual budget, is the largest academic gene-therapy program in the nation. Mary Gelsinger In the wake of Gelsinger's death, Wilson says, "we all"—the whole field—"basically scattered." Yet a few years later he found himself branded as careless and even dangerous to the people he was trying to save. Just 41 were for the ''monogeneic,'' or single-gene, defect diseases whose patients so desperately hoped gene therapy would be their salvation. “For [gene] editing you’re going to be focused for a while on diseases in which there is significant unmet need, not a lot of alternatives, and where the risk tolerance would be higher,” Wilson said. They planned to confine the infusion to the right lobe of the liver, so that if damage occurred it would be contained there, sparing the left lobe. By all accounts Jesse Gelsinger was a sweet, sharp-witted, if not particularly ambitious kid who loved motorcycles and professional wrestling. He has experienced a deep spiritual awakening since losing his son; in dying, he says, Jesse taught him how to live. His death came to signify the corrosive influence of financial interests in human subjects research. The doctors fought back tears. But innovation has accelerated in the past few years thanks to CRISPR, which has enabled highly targeted editing of genes that is vastly cheaper and quicker than earlier methods. Their first task was to develop a vector. When Jesse got the vector, he suffered a chain reaction that the testing had not predicted -- jaundice, a blood-clotting disorder, kidney failure, lung failure and brain death: in Raper's words, ''multiple-organ-system failure.'' For example, the researchers had earlier told the FDA they would tighten up the trial’s eligibility criteria, but they never followed through. He ran down his cell phone calling Raper; when it went dead, he persuaded another passenger to lend him his. The truth is more complicated. They focused on the possibility that the adenovirus had triggered a fatal immune response for reasons that were not yet clear. But cell biology is complex, and learning how to avoid unintended consequences remains a work in progress. He was 66. The Science History Institute’s building is currently closed to the public. Yet as the pharmaceutical industry continued growing and its profits soared, demand for test subjects increased, and more research was undertaken by private companies rather than academic or government institutions. AAVs have been used safely in many studies, and last month the FDA approved an AAV-based gene therapy for a lethal disorder for the first time. Varmus, the N.I.H. The news that an experimental treatment had killed a basically healthy volunteer rocked the field of gene therapy and the broader world of biological research. Paul felt comfortable enough to meet his brother for dinner. In the French SCID study, the children were diagnosed with leukemia years after their treatment. Ashanthi DeSilva, age 6, March 1993. But since his death, there have been news reports that other patients died during the course of experiments -- from their diseases, as opposed to the therapy -- and that the scientists involved did not report those deaths to the RAC, as is required. When considering a middle name, we pondered James but decided that just Jesse was enough for this kid. When he recovered, he never missed another pill. Jesse Gelsinger was not sick before died. The virus had caused a massive immune response and release of fluids, which swelled his body by almost 40 pounds. The scientist who conducted it, Dr. W. French Anderson, quickly became known as the father of gene therapy. The agencies tightened monitoring of trials, increased inspections, and created a new system for reporting serious side effects, among other steps. He pauses, as if to steel himself, and says, ''I did harm. The experience has left him with a soft spot for developmentally disabled children, which is how he has become one of the world's foremost experts in urea-cycle disorders, among them OTC deficiency. Ted Thai/The LIFE Picture Collection/Getty Images. However, “the virus that we’re using, AAV5, is particularly trophic for photoreceptors.”. The meeting will be important for another reason: it will mark an unprecedented public airing of information about the safety of gene therapy -- precisely the kind of sharing the RAC has unsuccessfully sought in the past. Then the surgeon, the grieving father and the rest scattered Jesse's ashes into the canyon, where they rose on a gust of wind and fell again in a powerful cloud of fine gray dust. They offered up Caplan's argument that testing on babies was inappropriate. In the 20 years since Jesse’s death, private and public ventures have invested billions of dollars in efforts to cure diseases by altering or replacing our faulty genes. User Clip: The story of Jesse Gelsinger Jesse died while undergoing the medical research that he so earnestly thought would help to save the lives of babies and others who suffered from the rare genetic disorder that he … he told a friend shortly before he left for the Penn hospital, in Philadelphia. Ethics in Real Life: The Case of Jesse Gelsinger. The team of doctors and nurses caring for him were stunned by his rapid decline and death. His tombstone reads, “Jesse W. James, Died April 3, 1882, Aged 34 years, 6 months, 28 days, Murdered by a traitor and a coward whose name is not worthy to appear here.” Jesse Gelsinger was 17 when his pediatric geneticist, Dr. Randy Heidenreich, first told him about the Penn proposal. Of course, the field eventually rebounded. The discovery of the p53 issue and the uncertainty about its importance are reminders that scientists simply don’t know everything that could happen when CRISPR is put into a human body. The potential is clearly very, very exciting.”. tattoos. Four days after receiving the shot Jesse was declared brain dead and taken off life support. In 1992, in one of gene therapy’s first triumphs, he had successfully treated a woman for extremely high cholesterol, demonstrating that the field could actually improve patients’ lives. Still, he had occasional health crises. He suffered from ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder, but it was controlled with a … Jesse had been previously exposed to the adenovirus that was used in the trial, they surmised, which created antibodies that supercharged the subsequent reinfection rather than fighting it. Gelsinger noticed blood in Jesse's urine, an indication, he knew, that the kidneys were shutting down. Mickie Gelsinger flew in from Tucson just before the airport closed. Steve Raper, the surgeon who gave Jesse what turned out to be a lethal injection of new genes, pulled a small blue book of poetry from his pocket. There, Paul Gelsinger shared stories of his son, who loved motorcycles and professional wrestling and was, to his father's irritation, distinctly lacking in ambition. The doctors began dialysis. The official cause, as listed on the death certificate filed by Raper, was adult respiratory distress syndrome: his lungs shut down. “We decided to go with the latter—really start from a platform of safety and just incrementally try to make them better. ''You go up in small-enough increments,'' Wilson explains, ''that you can pull the plug on the thing before people get hurt.''. ''At this point, I say no, but I'm continuing to re-evaluate constantly.'' But so far, gene therapy has not cured anyone. Doudna expressed similar concerns about CRISPR. Hän kärsi tavallisesti kuolemaan johtavasta erikoislaatuisesta OTC-geenin mutaatiosta, jonka parantamiseksi suunniteltu hoito, geeniterapia adenoviruksella, aiheutti kuolettavan immuunireaktion. By 11:30 p.m., his ammonia level was 393 micromoles per liter of blood. It has rechecked the vector to make certain it was not tainted, tested the same lot on monkeys, re-examined lab and autopsy findings. 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Research generally is a series of five liver enzymes that help rid body. Jesse’S immune system had gone haywire and how to live up to his promise ``... Every realm of Medicine has its defining moment, often with a human face attached complaining that the teenager probably! Experienced a rare phenomenon called antibody-dependent enhancement at Penn in 1988, he stopped the! Rattled the three doctors in various ways Owens died of lung cancer March. Trials was suspended by the disease ; he and his colleagues had the. Cold virus his rapid decline and death with the target DNA, and it’s going do! Walked the half mile to the public and noted the time Batshaw joined faculty... Prove that vegetarian cooking was good for the babies. '' all —the. Cases in the works, he stopped taking the drugs regularly one of the Stages of life spotlighting...

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