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

In a study published in Neuron, a research team at the Department of Neurology at Massachusetts General Hospital, aimed to ...

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

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

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 ...

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

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 ...

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 ...

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

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

Molecular biologists have long believed that the beginning of a gene launched the process of transcription—the process by ...

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