Renal Ciliopathies: Sorting Out Therapeutic Approaches for Nephronophthisis
Nephronophthisis (NPH) is an autosomal recessive ciliopathy and a major cause of end-stage renal disease in children.The main forms, juvenile and adult case of caymus NPH, are characterized by tubulointerstitial fibrosis whereas the infantile form is more severe and characterized by cysts.NPH is caused by mutations in over 20 different genes, most