{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T01:48:14Z","timestamp":1789523294063,"version":"build-2803163510"},"reference-count":92,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"7","license":[{"start":{"date-parts":[[2025,12,12]],"date-time":"2025-12-12T00:00:00Z","timestamp":1765497600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":["Kidney360"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:sec>\n                    <jats:title\/>\n                    <jats:p>\n                      Autosomal dominant polycystic kidney disease is the most common inherited kidney disorder and a leading monogenic cause of kidney failure. Reduction or loss of polycystin-1 and polycystin-2 function disrupts ciliary calcium signaling, elevates cyclic AMP (cAMP), reprograms cellular metabolism, and activates proliferative cascades that drive cyst expansion. Tolvaptan, a vasopressin V2 receptor antagonist, established cAMP modulation as a disease-modifying strategy but is limited by aquaretic effects and hepatotoxicity risk. This review highlights emerging therapeutic strategies in clinical trial development that extend beyond vasopressin antagonism. Gene-directed therapies aim to restore polycystin dosage, including anti\u2013miR-17 oligonucleotides (\n                      <jats:italic toggle=\"yes\">e.g<\/jats:italic>\n                      ., farabursen) and pharmacochaperones that rescue misfolded polycystin-1 and restore trafficking in select\n                      <jats:italic toggle=\"yes\">PKD1<\/jats:italic>\n                      missense variants (\n                      <jats:italic toggle=\"yes\">e.g<\/jats:italic>\n                      ., VX-407). Paracrine signaling can be modulated with anti\u2013pregnancy-associated plasma protein A antibodies that reduce insulin-like growth factor-1 bioavailability in cystic microenvironments. Metabolic reprogramming is targeted by agents such as metformin, bempedoic acid, glucagon-like peptide-1 receptor agonists, and structured dietary interventions. Sodium-glucose cotransporter 2 inhibitors hold theoretical promise but await definitive results from ongoing trials. A novel cAMP-lowering strategy through phosphodiesterase-4 activation is advancing toward clinical testing. Looking ahead, gene therapy, and genome editing offer the potential to raise polycystin levels above the threshold for cystogenesis, although challenges in vector capacity, kidney-specific delivery, and durability remain. Artificial intelligence\u2013guided discovery, coupled with human organoid platforms, is accelerating therapeutic repurposing and rational combination design. Collectively, these advances signal a transition toward a layered, mechanism-guided framework in which vasopressin blockade is integrated with metabolic, other signaling, and genotype-specific therapies. As biomarkers and risk stratification tools mature, autosomal dominant polycystic kidney disease management is poised to become increasingly precise, tolerable, and effective.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.34067\/kid.0000001109","type":"journal-article","created":{"date-parts":[[2025,12,12]],"date-time":"2025-12-12T17:00:25Z","timestamp":1765558825000},"page":"1662-1676","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":6,"title":["Emerging Therapies in Autosomal Dominant Polycystic Kidney Disease"],"prefix":"10.34067","volume":"7","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2726-3989","authenticated-orcid":false,"given":"Christopher Y.","family":"Chen","sequence":"first","affiliation":[{"name":"PKD Foundation, Kansas City, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6047-8792","authenticated-orcid":false,"given":"Mohamad","family":"Hadla","sequence":"additional","affiliation":[{"name":"Division of Nephrology and Hypertension, Mayo Clinic, Jacksonville, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ibrahim","family":"Khambati","sequence":"additional","affiliation":[{"name":"Division of Nephrology and Hypertension, Mayo Clinic, Jacksonville, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonu","family":"Kashyap","sequence":"additional","affiliation":[{"name":"Metabolism and Molecular Nutrition Laboratory, Kogod Center on Aging, The Glenn Foundation for Medical Research at the Mayo Clinic, Department of Anesthesiology and Perioperative Medicine, Mayo Clinic College of Medicine, Jacksonville, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-2338-6696","authenticated-orcid":false,"given":"Vanessa","family":"Westerfield","sequence":"additional","affiliation":[{"name":"PKD Foundation, Kansas City, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2725-4628","authenticated-orcid":false,"given":"Sorin","family":"Fedeles","sequence":"additional","affiliation":[{"name":"Critical Path Institute, Tucson, Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8283-1507","authenticated-orcid":false,"given":"Whitney","family":"Besse","sequence":"additional","affiliation":[{"name":"Section of Nephrology, Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2420-2358","authenticated-orcid":false,"given":"Katharina","family":"Hopp","sequence":"additional","affiliation":[{"name":"Division of Renal Diseases and Hypertension, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, Colorado"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5304-6593","authenticated-orcid":false,"given":"Peter C.","family":"Harris","sequence":"additional","affiliation":[{"name":"Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2875-4659","authenticated-orcid":false,"given":"Vishal","family":"Patel","sequence":"additional","affiliation":[{"name":"Division of Nephrology, Department of Internal Medicine, UT Southwestern Medical Center, Dallas, Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eduardo","family":"Chini","sequence":"additional","affiliation":[{"name":"Metabolism and Molecular Nutrition Laboratory, Kogod Center on Aging, The Glenn Foundation for Medical Research at the Mayo Clinic, Department of Anesthesiology and Perioperative Medicine, Mayo Clinic College of Medicine, Jacksonville, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4568-8967","authenticated-orcid":false,"given":"Mahdi","family":"Salih","sequence":"additional","affiliation":[{"name":"Division of Nephrology and Transplantation, Department of Internal Medicine, Erasmus Medical Center, Rotterdam, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8568-5664","authenticated-orcid":false,"given":"Michael A.","family":"Barry","sequence":"additional","affiliation":[{"name":"Division of Infectious Disease, Mayo Clinic, 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