内容摘要：of organ transplantation – and there’s no rolling around in the mud here.

of organ transplantation – and there’s no rolling around in the mud here.

’ Emmy Award. “I grabbed both my parents’ watches and my dad’s dress watch,” which she had given to her son Cooper for his 18th birthday.She also managed to pack a few pieces of clothing. “All our belongings are crammed into three tote bags, three large canvas bags, and several Macy’s plastic bags along with a CVS bag,” she said.

The fire completely devastated her property. “We lost everything. The only thing that survived is our front gate and the barbecue,” Rivers shared.She described the scale of destruction as unimaginable.“It’s so much more than just the homes — it’s an entire town wiped off the map,” she said. “Many businesses in Palisades were family-owned. There are people who not only lost their homes but also their businesses and livelihoods.”

Finding safe shelter has been another challenge.“We’ve had to move three times due to evacuations, but we’ve settled at a friend’s house in Santa Monica,” Rivers said. “They’ve been kind enough to take us in, along with another family we’re friends with.”

Rivers copes with these challenges through her self-proclaimed “dark sense of humor” and her knack for making people laugh. She also finds solace in supporting others who are struggling, which helps to ease her own burdens.

“Checking in and comforting the people in my world that are affected by this allows me to not deal with my home stuff right now. I cried once for five minutes and then again for like 20 and don’t have time to do that. I am my parents’ daughter.”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.