The head of the World Food Program, Cindy McCain, told ABC News that staffers on the ground were reporting people killed and called it a “tragedy.”
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 thatin 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.”. One seeks to create a registry of dog walkers who must pass an exam every two years and undergo CPR and animal behavior training.
Co-owner Rocio Dominguez offers a treat to Tony, one of her regulars, at Chumbis, an artisanal bakery for animals, in Buenos Aires, Argentina, Friday, April 4, 2025. (AP Photo/Natacha Pisarenko)Co-owner Rocio Dominguez offers a treat to Tony, one of her regulars, at Chumbis, an artisanal bakery for animals, in Buenos Aires, Argentina, Friday, April 4, 2025. (AP Photo/Natacha Pisarenko)
“I see an opportunity for it to become the most pet-friendly city in the region,” Ferrario said.Other politicians fret about the proliferation of pet-keeping as a symptom of a bigger crisis. They ask why young people in Argentina choose raising pets over raising children as the country ages rapidly.