First Personalized Gene Therapy Using Base Editing Shows Promising Results in Baby with Rare Disorder

Behind the Headlines, Episode 17: Bespoke CRISPR Therapy, Executive Order Fallout, and More

Jamie Baumgartner, Jonathan Grinstein, and John Wilkerson go behind the headlines to discuss the implications of personalized gene-editing therapies, more HHS policy and funding updates, and the latest tariff-related investments.

In the United States, a baby with a rare and life-threatening disease was treated 7 months after being born with a gene therapy specially designed for him. A big step towards personalized medicine analyzed by the General Manager of Genethon, Frédéric Revah.

Meet the 11-year-old Rowlett girl making medical history

11-year-old Marley Mansour receives the first-ever treatment for rare NARS1 disorder, offering hope for her and others affected by the condition.

Scientists Edited Genes Inside a Living Person for the First Time—and Saved His Life

With grim prognosis hanging overhead, doctors and scientists at universities and institutions across the U.S. worked tirelessly to develop the world’s first custom gene-editing therapy to save the life of a newborn.

Metro family embraces life with baby battling rare, fatal genetic disorder

OLATHE, Kan. — Five months ago, Christina and Adam Hannan were celebrating the arrival of their sixth child, a beautiful baby girl named Annalise. But the joy of her birth quickly turned to heartbreak. Annalise wasn’t crying. She wasn’t moving. Something was wrong. KC-area emergency management on staying prepared ahead of severe weather Doctors diagnosed her with congenital hypomyelinating neuropathy, a condition so rare that fewer than 50…

Gene editing helped a desperately ill Delaware County baby thrive. Scientists say it could someday treat millions

Researchers described the case in a new study, saying the baby is among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his genetic code that kills half of affected infants. A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, saying he’s among th…