Human and zebrafish data corroborate gene x gene interactions in ameliorating Duchenne muscular dystrophy disease progression

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder involving progressive muscle tissue loss with replacement by fat and fibrotic tissue, leading in most cases to loss of ambulation (LOA) by early to mid-adolescence. We have previously reported the largest DMD genome-wide analysis to date, using N = 419 patients from the United Dystrophinopathy Project (UDP) whose primary DMD mutations are presumed to preclude production of residual dystrophin. We found strong evidence for 6 modifiers of LOA, 4 of which have been validated in zebrafish. Here we follow up by investigating possib

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