{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T02:42:10Z","timestamp":1790649730957,"version":"4.1.0"},"reference-count":87,"publisher":"MDPI AG","issue":"2","license":[{"start":{"date-parts":[[2023,1,14]],"date-time":"2023-01-14T00:00:00Z","timestamp":1673654400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Autosomal dominant polycystic kidney disease, autosomal recessive polycystic kidney disease, and nephronophthisis are hereditary disorders with the occurrence of numerous cysts in both kidneys, often causing chronic and end-stage renal failure. Animal models have played an important role in recent advances in research not only on disease onset and progressive mechanisms but also on the development of therapeutic interventions. For a long time, spontaneous animal models have been used as the primary focus for human diseases; however, after the identification of the nucleotide sequence of the responsible genes, PKD1, PKD2, PKHD1, and NPHPs, various types of genetically modified models were developed by genetic and reproductive engineering techniques and played the leading role in the research field. In this review, we present murine models of hereditary renal cystic diseases, discussing their potential benefits in the development of therapeutic strategies.<\/jats:p>","DOI":"10.3390\/jcm12020668","type":"journal-article","created":{"date-parts":[[2023,1,16]],"date-time":"2023-01-16T04:31:32Z","timestamp":1673843492000},"page":"668","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Review of the Use of Animal Models of Human Polycystic Kidney Disease for the Evaluation of Experimental Therapeutic Modalities"],"prefix":"10.3390","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2014-9411","authenticated-orcid":false,"given":"Shizuko","family":"Nagao","sequence":"first","affiliation":[{"name":"Advanced Research Center for Animal Models of Human Diseases, Fujita Health University, Toyoake 470-1192, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tamio","family":"Yamaguchi","sequence":"additional","affiliation":[{"name":"Advanced Research Center for Animal Models of Human Diseases, Fujita Health University, Toyoake 470-1192, Japan"},{"name":"Department of Medical Technology, Faculty of Health Science, Suzuka University of Medical Science, Suzuka 510-0293, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,1,14]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Sekine, A., Hidaka, S., Moriyama, T., Shikida, Y., Shimazu, K., Ishikawa, E., Uchiyama, K., Kataoka, H., Kawano, H., and Kurashige, M. (2022). Cystic kidney diseases that require a differential diagnosis from autosomal dominant polycystic kidney disease (ADPKD). J. Clin. Med., 11.","DOI":"10.3390\/jcm11216528"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1159\/000506288","article-title":"Executive Summary: Clinical Practice Guideline for Autosomal Dominant Polycystic Kidney Disease in China","volume":"6","author":"Mei","year":"2020","journal-title":"Kidney Dis."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/S0140-6736(18)32782-X","article-title":"Autosomal dominant polycystic kidney disease","volume":"393","author":"Alam","year":"2019","journal-title":"Lancet"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"100887","DOI":"10.1016\/j.disamonth.2019.100887","article-title":"Update and review of adult polycystic kidney disease","volume":"66","author":"Colbert","year":"2020","journal-title":"Dis.-A-Mon."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1038\/s41572-018-0047-y","article-title":"Polycystic kidney disease","volume":"4","author":"Bergmann","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"key":"ref_6","doi-asserted-by":"crossref","unstructured":"Elisakova, V., Merta, M., Reiterova, J., Baxova, A., Kotlas, J., Hirschfeldova, K., Obeidova, L., Tesar, V., and Stekrova, J. (2018). Bilineal inheritance of pathogenic PKD1 and PKD2 variants in a Czech family with autosomal dominant polycystic kidney disease\u2014A case report. BMC Nephrol., 19.","DOI":"10.1186\/s12882-018-0978-2"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1649","DOI":"10.1038\/s41588-021-00946-4","article-title":"Renal plasticity revealed through reversal of polycystic kidney disease in mice","volume":"53","author":"Dong","year":"2021","journal-title":"Nat. Genet."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"1006","DOI":"10.1681\/ASN.2012070650","article-title":"Type of PKD1 Mutation Influences Renal Outcome in ADPKD","volume":"24","author":"Gall","year":"2013","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"962","DOI":"10.34067\/KID.0001022019","article-title":"Loss of Cilia Does Not Slow Liver Disease Progression in Mouse Models of Autosomal Recessive Polycystic Kidney Disease","volume":"1","author":"Gallagher","year":"2020","journal-title":"Kidney360"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"166348","DOI":"10.1016\/j.bbadis.2022.166348","article-title":"The genetics of Autosomal Recessive Polycystic Kidney Disease (ARPKD)","volume":"1868","author":"Goggolidou","year":"2022","journal-title":"Biochim. Biophys. Acta Mol. Basis Dis."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1072","DOI":"10.1542\/peds.111.5.1072","article-title":"Autosomal Recessive Polycystic Kidney Disease: The Clinical Experience in North America","volume":"111","author":"Desmond","year":"2003","journal-title":"Pediatrics"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"e74765","DOI":"10.7554\/eLife.74765","article-title":"A plasma membrane-localized polycystin-1\/polycystin-2 complex in endothelial cells elicits vasodilation","volume":"11","author":"MacKay","year":"2022","journal-title":"eLife"},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"31559","DOI":"10.1074\/jbc.M805452200","article-title":"Polycystin-2 Expression Is Regulated by a PC2-binding Domain in the Intracellular Portion of Fibrocystin","volume":"283","author":"Kim","year":"2008","journal-title":"J. Biol. Chem."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"1855","DOI":"10.1681\/ASN.2006121344","article-title":"Nephronophthisis-Associated Ciliopathies","volume":"18","author":"Hildebrandt","year":"2007","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_15","doi-asserted-by":"crossref","unstructured":"Malik, S.A., Modarage, K., and Goggolidou, P. (2020). The Role of Wnt Signalling in Chronic Kidney Disease (CKD). Genes, 11.","DOI":"10.3390\/genes11050496"},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"1158","DOI":"10.1177\/03009858211030541","article-title":"Histopathology is required to identify and characterize myopathies in high-throughput phenotype screening of genetically engineered mice","volume":"58","author":"Vogel","year":"2021","journal-title":"Vet. Pathol."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1210\/endrev\/bnaa033","article-title":"Mouse Models of Human Proprotein Convertase Insufficiency","volume":"42","author":"Shakya","year":"2021","journal-title":"Endocr. Rev."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"442","DOI":"10.24272\/j.issn.2095-8137.2021.387","article-title":"Characterization of two novel knock-in mouse models of syndromic retinal ciliopathy carrying hypomorphic Sdccag8 mutations","volume":"43","author":"Ren","year":"2022","journal-title":"Zool. Res."},{"key":"ref_19","doi-asserted-by":"crossref","unstructured":"Kim, J.H., Yu, J., Kim, H.K., Kim, J.Y., Kim, M.S., Cho, Y.G., Bae, S., Kang, K.K., and Jung, Y.J. (2022). Genome editing of golden SNP-carrying lycopene epsilon-cyclase (LcyE) gene using the CRSPR-Cas9\/HDR and geminiviral replicon system in rice. Int. J. Mol. Sci., 23.","DOI":"10.3390\/ijms231810383"},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1042\/BST20200757","article-title":"The cellular pathways and potential therapeutics of Polycystic Kidney Disease","volume":"49","author":"Richards","year":"2021","journal-title":"Biochem. Soc. Trans."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"18274","DOI":"10.1038\/s41598-021-97046-4","article-title":"Cystin genetic variants cause autosomal recessive polycystic kidney disease associated with altered Myc expression","volume":"11","author":"Yang","year":"2021","journal-title":"Sci. Rep."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"855","DOI":"10.1038\/ki.2012.446","article-title":"Macrophages promote polycystic kidney disease progression","volume":"83","author":"Vivian","year":"2013","journal-title":"Kidney Int."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"4506","DOI":"10.1172\/JCI128503","article-title":"Crystal deposition triggers tubule dilation that accelerates cystogenesis in polycystic kidney disease","volume":"129","author":"Torres","year":"2019","journal-title":"J. Clin. Investig."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"1505","DOI":"10.1093\/hmg\/ddn039","article-title":"Cyst formation and activation of the extracellular regulated kinase pathway after kidney specific inactivation of Pkd1","volume":"17","author":"Shibazaki","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1538\/expanim.61.477","article-title":"Animal Models for Human Polycystic Kidney Disease","volume":"61","author":"Nagao","year":"2012","journal-title":"Exp. Anim."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1007\/s11745-013-3859-2","article-title":"Renal Cyclooxygenase Products are Higher and Lipoxygenase Products are Lower in Early Disease in the pcy Mouse Model of Adolescent Nephronophthisis","volume":"49","author":"Yamaguchi","year":"2013","journal-title":"Lipids"},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"F557","DOI":"10.1152\/ajprenal.00299.2019","article-title":"Klotho supplementation ameliorates blood pressure and renal function in DBA\/2-pcy mice, a model of polycystic kidney disease","volume":"318","author":"Takenaka","year":"2020","journal-title":"Am. J. Physiol. Physiol."},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"3000","DOI":"10.2353\/ajpath.2010.100569","article-title":"Transgenic Overexpression of Anks6(p.R823W) Causes Polycystic Kidney Disease in Rats","volume":"177","author":"Neudecker","year":"2010","journal-title":"Am. J. Pathol."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"F1078","DOI":"10.1152\/ajprenal.00504.2009","article-title":"Polycystic kidney disease in Han:SPRD Cy rats is associated with elevated expression and mislocalization of SamCystin","volume":"299","author":"Nagao","year":"2010","journal-title":"Am. J. Physiol. Physiol."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1177\/030098588902600302","article-title":"Hereditary Polycystic Kidney Disease Associated with Osteorenal Syndrome in Rats","volume":"26","author":"Rapp","year":"1989","journal-title":"Vet. Pathol."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"1033","DOI":"10.1007\/s40620-021-01160-5","article-title":"Novel 3D capsule device to restrict kidney volume expansion on polycystic kidney progression: Feasibility study in a rat model","volume":"35","author":"Bae","year":"2021","journal-title":"J. Nephrol."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1681\/ASN.2007060688","article-title":"Vasopressin Directly Regulates Cyst Growth in Polycystic Kidney Disease","volume":"19","author":"Wang","year":"2007","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"1279","DOI":"10.1002\/ar.21166","article-title":"Disease Stage Characterization of Hepatorenal Fibrocystic Pathology in the PCK Rat Model of ARPKD","volume":"293","author":"Mason","year":"2010","journal-title":"Anat. Rec."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"1605","DOI":"10.1016\/S0002-9440(10)64116-8","article-title":"Polycystic Kidney Rat Is a Novel Animal Model of Caroli\u2019s Disease Associated with Congenital Hepatic Fibrosis","volume":"158","author":"Sanzen","year":"2001","journal-title":"Am. J. Pathol."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"1212","DOI":"10.2215\/CJN.05281107","article-title":"Role of Vasopressin Antagonists","volume":"3","author":"Torres","year":"2008","journal-title":"Clin. J. Am. Soc. Nephrol."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"41","DOI":"10.2147\/IJNRD.S125942","article-title":"Tolvaptan in the treatment of autosomal dominant polycystic kidney disease: Patient selection and special considerations","volume":"11","author":"Joly","year":"2018","journal-title":"Int. J. Nephrol. Renov. Dis."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"2220","DOI":"10.1681\/ASN.2006030251","article-title":"Increased Water Intake Decreases Progression of Polycystic Kidney Disease in the PCK Rat","volume":"17","author":"Nagao","year":"2006","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_38","doi-asserted-by":"crossref","unstructured":"Wang, H., Cui, J., Yang, C., Rosenblum, J.S., Zhang, Q., Song, Q., Pang, Y., Fang, F., Sun, M., and Dmitriev, P. (2019). A Transgenic Mouse Model of Pacak\u2013Zhuang Syndrome with An Epas1 Gain-of-Function Mutation. Cancers, 11.","DOI":"10.3390\/cancers11050667"},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1038\/s41581-018-0059-6","article-title":"Renal disease in tuberous sclerosis complex: Pathogenesis and therapy","volume":"14","author":"Lam","year":"2018","journal-title":"Nat. Rev. Nephrol."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"2552","DOI":"10.1038\/s41418-020-0521-4","article-title":"53BP1 loss rescues embryonic lethality but not genomic instability of BRCA1 total knockout mice","volume":"27","author":"Chen","year":"2020","journal-title":"Cell Death Differ."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1016\/j.coph.2009.06.017","article-title":"In vivo target validation using gene invalidation, RNA interference and protein functional knockout models: It is the time to combine","volume":"9","author":"Vidalin","year":"2009","journal-title":"Curr. Opin. Pharmacol."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"615491","DOI":"10.3389\/fgene.2021.615491","article-title":"Advances in genome editing and application to the generation of genetically modified rat models","volume":"12","author":"Chenouard","year":"2021","journal-title":"Front. Genet."},{"key":"ref_43","doi-asserted-by":"crossref","unstructured":"Zhao, L., Zhao, J., Zhang, Y., Wang, L., Zuo, L., Niu, A., Zhang, W., Xue, X., Zhao, S., and Sun, C. (2021). Generation and identification of a conditional knockout allele for the PSMD11 gene in mice. BMC Dev. Biol., 21.","DOI":"10.1186\/s12861-020-00233-1"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"00337-17","DOI":"10.1128\/MCB.00337-17","article-title":"Mitochondrial Abnormality Facilitates Cyst Formation in Autosomal Dominant Polycystic Kidney Disease","volume":"37","author":"Ishimoto","year":"2017","journal-title":"Mol. Cell. Biol."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"6493","DOI":"10.1096\/fj.201901739RR","article-title":"Increased mitochondrial fragmentation in polycystic kidney disease acts as a modifier of disease progression","volume":"34","author":"Cassina","year":"2020","journal-title":"FASEB J."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"154258","DOI":"10.1016\/j.metabol.2020.154258","article-title":"Targeting mammalian serine\/threonine-protein kinase 4 through Yes-associated protein\/TEA domain transcription factor-mediated epithelial-mesenchymal transition ameliorates diabetic nephropathy orchestrated renal fibrosis","volume":"108","author":"Yang","year":"2020","journal-title":"Metabolism"},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"1659","DOI":"10.1681\/ASN.2008030259","article-title":"Triptolide Reduces Cystogenesis in a Model of ADPKD","volume":"19","author":"Leuenroth","year":"2008","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"16640","DOI":"10.1038\/s41598-019-53061-0","article-title":"Polycystin-1 Regulates Actomyosin Contraction and the Cellular Response to Extracellular Stiffness","volume":"9","author":"Nigro","year":"2019","journal-title":"Sci. Rep."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"8876","DOI":"10.1111\/jcmm.15512","article-title":"Reducing YAP expression in Pkd1 mutant mice does not improve the cystic phenotype","volume":"24","author":"Formica","year":"2020","journal-title":"J. Cell. Mol. Med."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"629","DOI":"10.1002\/med.21850","article-title":"Autosomic dominant polycystic kidney disease and metformin: Old knowledge and new insights on retarding progression of chronic kidney disease","volume":"42","author":"Casarella","year":"2021","journal-title":"Med. Res. Rev."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"2529","DOI":"10.1681\/ASN.2021010101","article-title":"Integrative Cistromic and Transcriptomic Analyses Identify CREB Target Genes in Cystic Renal Epithelial Cells","volume":"32","author":"Liu","year":"2021","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"e15058","DOI":"10.14814\/phy2.15058","article-title":"\u03b23 adrenergic receptor as potential therapeutic target in ADPKD","volume":"9","author":"Schena","year":"2021","journal-title":"Physiol. Rep."},{"key":"ref_53","doi-asserted-by":"crossref","unstructured":"M\u00e9ndez-Barbero, N., Guti\u00e9rrez-Munoz, C., Bl\u00e1zquez-Serra, R., Mart\u00edn-Ventura, J.L., and Blanco-Colio, L.M. (2020). Tumor necrosis factor-like weak inducer of apoptosis (TWEAK)\/fibroblast growth factor-inducible 14 (Fn14) axis in cardiovascular diseases: Progress and challenges. Cells, 9.","DOI":"10.3390\/cells9020405"},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"1913","DOI":"10.1681\/ASN.2020071094","article-title":"TWEAK Signaling Pathway Blockade Slows Cyst Growth and Disease Progression in Autosomal Dominant Polycystic Kidney Disease","volume":"32","author":"Cordido","year":"2021","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"179","DOI":"10.34067\/KID.0001052019","article-title":"Interferon Regulatory Factor-5 in Resident Macrophage Promotes Polycystic Kidney Disease","volume":"1","author":"Zimmerman","year":"2020","journal-title":"Kidney360"},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1172\/JCI76972","article-title":"Polycystin-1 maturation requires polycystin-2 in a dose-dependent manner","volume":"125","author":"Gainullin","year":"2015","journal-title":"J. Clin. Investig."},{"key":"ref_57","first-page":"e141299","article-title":"Ciclopirox olamine induces ferritinophagy and reduces cyst burden in polycystic kidney disease","volume":"6","author":"Radadiya","year":"2021","journal-title":"J. Clin. Investig."},{"key":"ref_58","doi-asserted-by":"crossref","unstructured":"Zhou, J., and Li, X. (2021). Non-Coding RNAs in Hereditary Kidney Disorders. Int. J. Mol. Sci., 22.","DOI":"10.3390\/ijms22063014"},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"e133785","DOI":"10.1172\/jci.insight.133785","article-title":"Interstitial microRNA miR-214 attenuates inflammation and polycystic kidney disease progression","volume":"5","author":"Lakhia","year":"2020","journal-title":"JCI Insight"},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"4148","DOI":"10.1038\/s41467-019-11918-y","article-title":"Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease","volume":"10","author":"Lee","year":"2019","journal-title":"Nat. Commun."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"1697","DOI":"10.1681\/ASN.2020020190","article-title":"Epithelial Vasopressin Type-2 Receptors Regulate Myofibroblasts by a YAP-CCN2\u2013Dependent Mechanism in Polycystic Kidney Disease","volume":"31","author":"Dwivedi","year":"2020","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"1200","DOI":"10.1038\/s42003-021-02730-w","article-title":"Metabolic derangement in polycystic kidney disease mouse models is ameliorated by mitochondrial-targeted antioxidants","volume":"4","author":"Daneshgar","year":"2021","journal-title":"Commun. Biol."},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"906","DOI":"10.1016\/j.kint.2021.12.006","article-title":"Elamipretide treatment during pregnancy ameliorates the progression of polycystic kidney disease in maternal and neonatal mice with PKD1 mutations","volume":"101","author":"Daneshgar","year":"2021","journal-title":"Kidney Int."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"F27","DOI":"10.1152\/ajprenal.00298.2021","article-title":"Metformin improves relevant disease parameters in an autosomal dominant polycystic kidney disease mouse model","volume":"322","author":"Li","year":"2022","journal-title":"Am. J. Physiol. Physiol."},{"key":"ref_65","first-page":"e21874","article-title":"Pre-clinical evaluation of dual targeting of the GPCRs CaSR and V2R as therapeutic strategy for autosomal dominant polycystic kidney disease","volume":"35","author":"Wang","year":"2021","journal-title":"FASEB J."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"1087","DOI":"10.1681\/ASN.2021081125","article-title":"Protein Kinase A Downregulation Delays the Development and Progression of Polycystic Kidney Disease","volume":"33","author":"Wang","year":"2022","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"e21987","DOI":"10.1096\/fj.202101315R","article-title":"VX-809 mitigates disease in a mouse model of autosomal dominant polycystic kidney disease bearing the R3277C human mutation","volume":"35","author":"Yanda","year":"2021","journal-title":"FASEB J."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"947","DOI":"10.1038\/s41419-021-04248-9","article-title":"The tyrosine-kinase inhibitor Nintedanib ameliorates autosomal-dominant polycystic kidney disease","volume":"12","author":"Jamadar","year":"2021","journal-title":"Cell Death Dis."},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1681\/ASN.2013121312","article-title":"Tolvaptan plus Pasireotide Shows Enhanced Efficacy in a PKD1 Model","volume":"26","author":"Hopp","year":"2015","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"195","DOI":"10.3892\/mmr.2021.11834","article-title":"Combination of curcumin and ginkgolide B inhibits cystogenesis by regulating multiple signaling pathways","volume":"23","author":"Li","year":"2021","journal-title":"Mol. Med. Rep."},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"104751","DOI":"10.1016\/j.phrs.2020.104751","article-title":"Cardamonin retards progression of autosomal dominant polycystic kidney disease via inhibiting renal cyst growth and interstitial fibrosis","volume":"155","author":"He","year":"2020","journal-title":"Pharmacol. Res."},{"key":"ref_72","doi-asserted-by":"crossref","first-page":"e22089","DOI":"10.1096\/fj.202101270RR","article-title":"Antioxidant enzyme peroxiredoxin 5 regulates cyst growth and ciliogenesis via modulating Plk1 stability","volume":"36","author":"Agborbesong","year":"2022","journal-title":"FASEB J."},{"key":"ref_73","first-page":"e135385","article-title":"Polycystin-1 regulates ARHGAP35-dependent centrosomal RhoA activation and ROCK signaling","volume":"5","author":"Streets","year":"2020","journal-title":"J. Clin. Investig."},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"2384","DOI":"10.1080\/15548627.2020.1826716","article-title":"Loss of PKD1\/polycystin-1 impairs lysosomal activity in a CAPN (calpain)-dependent manner","volume":"17","author":"Peintner","year":"2021","journal-title":"Autophagy"},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"14443","DOI":"10.1038\/s41598-021-93633-7","article-title":"Smoking accelerates renal cystic disease and worsens cardiac phenotype in Pkd1-deficient mice","volume":"11","author":"Sousa","year":"2021","journal-title":"Sci. Rep."},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"4320","DOI":"10.1038\/s41467-020-18104-5","article-title":"Cyst growth in ADPKD is prevented by pharmacological and genetic inhibition of TMEM16A in vivo","volume":"11","author":"Cabrita","year":"2020","journal-title":"Nat. Commun."},{"key":"ref_77","first-page":"e146041","article-title":"Restoration of proximal tubule flow\u2013activated transport prevents cyst growth in polycystic kidney disease","volume":"6","author":"Du","year":"2021","journal-title":"J. Clin. Investig."},{"key":"ref_78","doi-asserted-by":"crossref","first-page":"2319","DOI":"10.1681\/ASN.2015060634","article-title":"MicroRNA-21 Aggravates Cyst Growth in a Model of Polycystic Kidney Disease","volume":"27","author":"Lakhia","year":"2016","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_79","first-page":"e135700","article-title":"Metalloproteinase PAPP-A regulation of IGF-1 contributes to polycystic kidney disease pathogenesis","volume":"5","author":"Kashyap","year":"2020","journal-title":"J. Clin. Investig."},{"key":"ref_80","doi-asserted-by":"crossref","first-page":"2159","DOI":"10.1681\/ASN.2020111560","article-title":"Inhibiting Focal Adhesion Kinase Ameliorates Cyst Development in Polycystin-1\u2013Deficient Polycystic Kidney Disease in Animal Model","volume":"32","author":"He","year":"2021","journal-title":"J. Am. Soc. Nephrol."},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1016\/j.cmet.2021.03.024","article-title":"A methionine-Mettl3-N(6)-methyladenosine axis promotes polycystic kidney disease","volume":"33","author":"Ramalingam","year":"2021","journal-title":"Cell Metab."},{"key":"ref_82","doi-asserted-by":"crossref","first-page":"eaba3613","DOI":"10.1126\/scitranslmed.aba3613","article-title":"Activation of NRF2 ameliorates oxidative stress and cystogenesis in autosomal dominant polycystic kidney disease","volume":"12","author":"Lu","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"ref_83","doi-asserted-by":"crossref","first-page":"4548","DOI":"10.1038\/s41467-021-24799-x","article-title":"Extracellular vesicles and exosomes generated from cystic renal epithelial cells promote cyst growth in autosomal dominant polycystic kidney disease","volume":"12","author":"Ding","year":"2021","journal-title":"Nat. Commun."},{"key":"ref_84","doi-asserted-by":"crossref","first-page":"F258","DOI":"10.1152\/ajprenal.00262.2021","article-title":"Metabolic reprogramming in a slowly developing orthologous model of polycystic kidney disease","volume":"322","author":"Hopp","year":"2022","journal-title":"Am. J. Physiol. Physiol."},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"F463","DOI":"10.1152\/ajprenal.00181.2018","article-title":"Heterozygous Pkhd1C642* mice develop cystic liver disease and proximal tubule ectasia that mimics radiographic signs of medullary sponge kidney","volume":"316","author":"Shan","year":"2019","journal-title":"Am. J. Physiol. Physiol."},{"key":"ref_86","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1093\/hmg\/ddab239","article-title":"An Nphp1 knockout mouse model targeting exon 2\u201320 demonstrates characteristic phenotypes of human nephronophthisis","volume":"31","author":"Li","year":"2021","journal-title":"Hum. Mol. Genet."},{"key":"ref_87","doi-asserted-by":"crossref","first-page":"e3082","DOI":"10.1002\/jgm.3082","article-title":"Creating cell and animal models of human disease by genome editing using CRISPR\/Cas9","volume":"21","author":"Zarei","year":"2019","journal-title":"J. Gene Med."}],"container-title":["Journal of Clinical Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2077-0383\/12\/2\/668\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T18:05:59Z","timestamp":1760119559000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2077-0383\/12\/2\/668"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,1,14]]},"references-count":87,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2023,1]]}},"alternative-id":["jcm12020668"],"URL":"https:\/\/doi.org\/10.3390\/jcm12020668","relation":{},"ISSN":["2077-0383"],"issn-type":[{"value":"2077-0383","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,1,14]]}}}