Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
Amaurosis congenita of Leber, type 1
0.020 GeneticVariation disease BEFREE Phase I clinical trials to cure LCA and choroideremia demonstrated that adeno-associated viral vectors containing RPE genes and photoreceptors respectively, could be successfully administered to inherited retinopathy patients. 28393043 2017
Amaurosis congenita of Leber, type 1
0.020 AlteredExpression disease BEFREE S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA. 23335596 2013