Martin, 20, was the first patient in St. Louis and among the first across the country to receive a new gene therapy for sickle ... and friends made — full of pictures, encouraging notes and ...

Color vision evolved to support survival and social needs. Today, it influences how we think, feel, and navigate the world, ...

She arrived to find the screens in the studio filled with images ... gene that seems to play such an important role in pain processing makes it a particularly attractive target for gene therapy ...

Rapid technological advancements are increasingly narrowing the line between humans and machines. At the forefront of this ...

A team of doctors at the Children's Hospital of Philadelphia and Penn Medicine treated a baby with a gene editing technology ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to a well-tolerated safety profile, which has been heralded by analysts as ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects ...

A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized ...

The patient received 3E15 vector genomes of gene therapy NGN-401 on Nov. 5 as part of the high-dose arm of Neurogene’s ongoing phase 1/2 open-label, pediatric study. She developed a fever before ...

This technique streamlines quality control in gene therapy vector production, potentially accelerating manufacturing and improving clinical outcomes. This summary was automatically generated using ...

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...

For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of the missing genes to the affected cells. Despite those efforts ...