02Sep/13

Argininosuccinic aciduria (ASA) and gene therapy: an interview with Dr Julien … – News-Medical.net

Argininosuccinic aciduria (ASA) and gene therapy: an interview with Dr Julien
News-Medical.net
To “repair” a genetically damaged liver urea cycle, liver transplantation (LT) can be used as treatment for ASA. However, LT carries a risk of mortality, long term complications, and a life-long immunosuppression. In addition there is worldwide acute