Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
Granulomatous Disease, Chronic, X-Linked
0.020 Biomarker disease BEFREE Because the success of retroviral gene therapy for X-CGD has been hampered by insertional activation of proto-oncogenes, targeting the insertion of a gp91phox transgene into potential safe harbor sites, such as AAVS1, may represent a valid alternative. 26295532 2015
Granulomatous Disease, Chronic, X-Linked
0.020 GeneticVariation disease BEFREE Zinc finger nuclease-mediated gene targeting of a single-copy gp91(phox) therapeutic minigene into one allele of the "safe harbor" AAVS1 locus in X-CGD iPSCs without off-target inserts resulted in sustained expression of gp91(phox) and substantially restored neutrophil ROS production. 21411759 2011