Edging closer to successful gene therapy for Wilson disease

Abstract

In a recent article in Molecular Therapy – Methods and Clinical Development, Padula and co-authors present pre-clinical data for a new approach to gene therapy in Wilson disease (WD).1 By using dual adeno-associated viral (AAV) vectors combined with split-intein technology to catalyze ligation of two separate peptides, the authors manage to transfect a full-size ATP7B protein to a mouse model of WD. The pre-clinical data from the present study are encouraging and pave the way for future clinical studies.