KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

KJ Muldoon, an infant with a life-threatening metabolic disorder who received a pioneering gene-editing therapy at Children’s Hospital of Philadelphia, went home Tuesday after 307 days at the hospital ...

After 307 days at the Children's Hospital of Philadelphia, a local baby who was born with a rare and usually fatal disorder ...

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

After 307 days at CHOP, KJ was dressed in what early studies suggest are the world’s smallest and cutest graduation ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

This study reveals the potential of engineered phage-IL-12 therapy in melanoma, enhancing immune response and tumor ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...