Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
Amaurosis congenita of Leber, type 1
0.010 AlteredExpression disease BEFREE Subretinal delivery of AAV5-IRBP/GNAT2-hDIO3 induced robust expression of DIO3 in the mouse retina and significantly reduced the number of TUNEL-positive cells in the cone-dominant LCA model Rpe65 <sup>-/-</sup> /Nrl <sup>-/-</sup> mice. 29721936 2018