A vast majority of orphan diseases are of genetic origin. Recent technological advances now permit the routine identification of causative mutations in genetic disease and offer the potential to repair genetic defects in a therapeutic context. This group focuses on the initial identification of novel genes and mutations in familial autosomal recessive and dominant orphan disease in Austria. Candidate disease-causing alterations will be used to develop novel, tailored, pre-clinical, genome editing based autologous cellular therapy transplantation strategies.
We utilize next generation sequencing, bioinformatics platforms, autologous precursor cell culture systems and both adenoviral and nonviral gene transfer technologies.
Kontoverbindung/Account details
Medizinische Universität Wien/Medical University of Vienna
Genetik seltener Erkrankungen/Donations genetics of rare diseases
Erste Bank
Adress/Address: Alserstraße 23, 1080 Wien
IBAN: AT362011140410070700
BIC: GIBAATWWXX
Kennzahl/Reference number UE61301002 (bitte unbedingt eintragen/please always include)