The U.S. has one of the

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

, for example, treats sickle cell disease, a painful blood disorder affecting millions worldwide.Musunuru said his team’s work —— 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.”An Israeli offensive launched after the attack has killed more than 51,000 Palestinians, more than half of them women and children, according to local health authorities, and reduced large parts of Gaza to rubble. U.S.-led efforts to broker a ceasefire and bring home remaining hostages appear to be at a standstill.

Nir Oz was one of the, with nearly a quarter of the approximately 400 residents killed or captured in the 2023 attack. For Israelis, it stands out as the embodiment of their

that day. Soldiers took hours to respond. Some families have said they saw Hamas militants killing or kidnapping animals.Rachel Dancyg holds her dog, Billie, on Wednesday, April 16, 2025. Israeli soldiers found Billie in the Gaza Strip, 18 months after she disappeared during Hamas’ attack on Dancyg’s Kibbutz of Nir Oz, in Binyamina, Israel. (AP Photo/Ariel Schalit)