Determination of the role of SF3B1 in the regulation of the adeno-associated virus life cycle. KU Leuven
We study a virus, called adeno-associated virus (AAV), that can be engineered to transfer therapeutic genes to patients with genetic diseases such as haemophilia. Despite therapeutic success, a number of questions regarding the biology of this virus remain. For example, it is not known if viral vectors which mimic the latent phase of the viral life cycle, like their natural counterparts, undergo silencing once they are inside the body. Here ...