Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

In gene therapy techniques, a harmless virus called the Adeno-Associated Virus (AAV) is modified to deliver the therapeutic genes into cells at the back of the eye, helping them to function ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...

Until recently, some eye diseases had been considered incurable but new gene therapy techniques have shown great promise. However, increasing evidence from clinical trials have reported problems ...

Researchers at Kumamoto University have successfully executed a practical trial of a new genome-editing technique that allows ...

In this week’s edition of the biotech bi-weekly, learn more about CRAIC technologies’ 5D Spectral Surface Mapping solution, ...

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene therapy, data that are prompting the biotech to seek accelerated approval ...

Regeneron’s investigational gene therapy has been tied to notable hearing improvements in 10 of 11 children who were treated for a rare genetic condition that causes hearing loss. “You can see ...