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. 2017 Mar 13;33(4):553–571. doi: 10.1007/s00467-017-3629-0

Table 2.

Evidence from animal and human studies of genes potentially involved in the pathogenesis of pelvi-ureteric junction obstruction

Gene Full gene name Animal Features and mechanism Human Reference
Ace Angiotensin converting enzyme Ace −/− mice Hydronephrosis, renal parenchymal atrophy [51]
Adamts-1 A disintegrin-like and metallopeptidase with thrombospondin type 1 motif, 1 Adamts -1−/− mice PUJ obstruction, increased collagen at PUJ. Other urogenital anomalies. [52]
Agt Angiotensin Agt −/− mice Hydronephrosis, renal parenchymal atrophy, [53]
Agtr 1a/b Angiotensin II receptor type 1 (1a and 1b) Agtr1 −/− (1a and 1b) mice Hydronephrosis in older mice, renal parenchymal atrophy, failure of renal pelvis development, ureteric smooth muscle hypoplasia and abnormal peristalsis [31]
Aqp2 Aquaporin 2 Aqp 2S256L/S256L CPH mice Mutation in CPH mice prevents Aqp2 phosphorylation and normal trafficking. Hydronephrosis secondary to polyuria [54]
Calcineurin Calcineurin. Also known as Protein phosphatase 3 (ppp3) Pax3-Cre T/+; Cnb1 flox/ flox mice Calcineurin inactivation in metanephric and ureteral mesenchyme giving hydronephrosis, abnormal pyeloureteral peristalsis with defective renal pelvis and smooth muscle development [30]
Id2 Inhibitor of DNA binding 2 Id2 −/− and Id2 +/− mice Hydronephrosis and PUJ development [38]
Nfia Nuclear factor I/A Nfia +/− and Nfia −/− mice Hydroureteronephrosis, VUR, abnormal PUJ and VUJ development. CNS malformations. Nfia +/−due to chromosomal translocation and deletion. VUR and CNS malformations. [55]
TBX18 T-box transcription factor Tbx18 −/− mice Hydroureteronephrosis, short ureters, ureteric smooth muscle defects due to abnormal smooth muscle cell differentiation and localisation Hispanic family with autosomal dominant CAKUT predominantly PUJO. Heterozygous truncating mutation (c.1010delG) of Tbx18 [24, 56]
Tshz2 and 3 Teashirt zinc finger family member 2 and 3 Tshz3 −/− mice Hydronephrosis with PUJ configuration, abnormal smooth muscle differentiation proximal ureter Tshz2/Tshz3 mutations not cause of PUJO in Albanian/Macedonian population [57, 58]

CAKUT, Congenital anomalies of the kidney and urinary tract; CNS, central nervous system; CPH, congenital progressive hydronephrosis; PUJO, pelvi-ureteric junction obstruction; VUJ, vesico-ureteric junction; VUR, vesico-ureteric reflux