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

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Figure 1: Diagram of the anagen hair follicle ... a characteristic which could be exploited by in vivo and ex vivo gene therapy to produce molecules other than those involved directly in hair ...

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