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

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

Researchers have identified a new genetic cause of hereditary optic atrophy—a condition that leads to gradual vision loss—by discovering a previously unknown mutation in the PPIB gene. T ...

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 ...

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

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

A new study has identified a mutation in the TREM2 gene that disrupts the brain’s ability to clear toxic amyloid plaques, increasing Alzheimer’s disease risk.

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

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