内容摘要：Vaccination isn’t required in Mexico. Schools can request vaccination records, federal health department spokesman Carlos Mateos said, but they cannot deny anyone access to education.

Vaccination isn’t required in Mexico. Schools can request vaccination records, federal health department spokesman Carlos Mateos said, but they cannot deny anyone access to education.

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

, for example, treats sickle cell disease, a painful blood disorder affecting millions worldwide.

Musunuru said his team’s work —But Wang pointed to a potential next step in xenotransplantation — learning to transplant pig livers. His team reported Wednesday in the journal Nature that a pig liver transplanted into a brain-dead person survived for 10 days, with no early signs of rejection. He said the pig liver produced bile and albumin — important for basic organ function — although not as much as human livers do.

The liver is a complex challenge because of its varied jobs, including removing waste, breaking down nutrients and medicines, fighting infection, storing iron and regulating blood clotting.“We do find that it could function a little bit in a human being,” Wang said. He speculated that would be enough to help support a failing human liver.

In the U.S. last year, surgeons at the University of Pennsylvania attempted that sort of “bridge” supportto a brain-dead human body to filter blood, much like dialysis for failing kidneys. U.S. pig developer eGenesis is studying that approach.