SKY-AS

A therapeutic approach where a healthy copy of a virus, Adeno-Associated Virus (AAV), is used to carry a healthy copy of a gene to a target organ. This is most commonly delivered in-vivo (inside the body). For Angelman syndrome (AS) the healthy copy of the missing or non-functional UBE3A gene is packaged inside the AAV, and is injected into the fluid that surrounds the brain, called the cerebrospinal fluid (CSF). Once in the fluid, the virus and gene can directly reach important cells of the brain, called neurons, and replace the non-functional or missing copy of the gene.