Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...

At Mayo Clinic, cardiologists Peter Noseworthy, M.D., and John Giudicessi, M.D., Ph.D., are uncovering the earliest signs of ...

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...

Mother Daphne Graskewicz-Prado of Southern Illinois told Newsweek: “I wasn’t going to accept the terminal diagnosis.” ...

GATC brought together a broad coalition of researchers and executives in cell and gene therapy, gene editing, drug discovery and AI as well as patients and advocates.

On her mother’s instruction, a toddler gives her pink stuffed horse, Snap Snap, a big cuddle. Then, as told, she plants a kiss on his soft fur and, because he’s hungry, feeds him a pretend bowl of ...

Although rare kidney diseases remain a significantly overlooked contributor to the global burden of CKD, things have begun to ...

The research will support the implementation of precision medicine efforts for the population. Read more at straitstimes.com. Read more at straitstimes.com.

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...