DEVELOPMENTS MINIGENES FOR USH 2C

Erwin van Wijk will started ‘mini genes’ research in early 2020 for USH2C, titled ’Pre-clinical development of a minigene augmentation therapy for the future treatment of USH2C-associated retinitis pigmentosa’.

In the highly promising preliminary investigation with the USH2A Minigenes, which has demonstrated that it is possible to make minigenes and that these work in the correct/expected way.

In this research, a translation will be made to humans using human skin cells. In this way they can investigate whether the minigenes behave well in human cells and whether the proteins properly unfold.