Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

Researchers in Göttingen, Germany, have elucidated the structure and function of otoferlin, a protein that plays a crucial ...

A groundbreaking gene therapy has successfully restored immune function in children with ADA-SCID, a severe genetic disorder.

Scientists have now created an encyclopedia of gene function for all known human genes that code for protein. This data uses the most current and reliable data, and it has also been released on a ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

Johns Hopkins researchers have developed a powerful new AI tool called Splam that can identify where splicing occurs in genes—an advance that could help scientists analyze genetic data with greater ...

The gene therapy for neurological diseases market is poised for growth due to rising neurological disorder prevalence, demand ...

Dominika Pilat, PhD, and Ana Griciuc, PhD, of the Department of Neurology at Massachusetts General Hospital are the lead and senior authors of a paper published in Neuron, "The Gain-of-Function ...

Despite rapid advances in reading the genetic code of living organisms, scientists still face a major challenge today—knowing ...