Infant With Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment

Personalized gene editing saved a baby, but the tech’s future is uncertain

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

'Breathtaking' CRISPR gene treatment creates hope for rare diseases

Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this technique help to unlock treatments for others?

This Baby’s One-in-a-Million Genetic Disorder Had No Cure. So Scientists Designed One Just for Him

The first personalized CRISPR therapy saved a child’s life. Can it save others too?

Infant With Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children’s Hospital of Philadelphia and Penn Medicine. The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase deficiency (CPS1). After spending […] The post Infant With Incurable Disease is First to Successfully Receive Personaliz…

The future of technology seems uncertain after the successful use of personalized gene editing to save a baby's life

The announcement that a baby born in Philadelphia had the first gene therapy specifically designed for him was met with widespread approval, as it...

Scientists have developed a revolutionary new genetic editing system capable of integrating whole genes into human DNA. This could, in the long run, offer a concrete hope for patients suffering from complex genetic diseases caused by a multitude of different mutations. Up to now, genetic editing tools were often limited to correcting one or a few of the [...]