Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
CUI: C4721769
Disease: Citrullinemia Type 1
Citrullinemia Type 1
0.010 Biomarker disease BEFREE A murine model of CTLN1 (fold/fold) that displays lethality within the first 21 days of life was used to determine the efficacy of adeno-associated viral (AAV) gene transfer as a potential therapy. 24131980 2013