Gene therapy for genetic muscle and blood disorders

Several specific gene therapy applications are being developed with adenovirus vectors. Firstly, the Heart-pace group is involved in an international consortium that aims to express different ion channels in patient hearts to restore healthy pacemaker activity. Secondly, large libraries of adenovirus vectors are being studied on different leukocyte populations in the blood, including haematopoietic stem and NK cells, to improve therapies targeting these cell types that are still difficult to permanently alter. Thirdly, adenovirus vectors expressing the von Willebrand factor are being constructed for the treatment of coagulation disorders.

• Responsible: Chair of Virology and Microbiology