DeCure for Congenital anomalies of kidney and urinary tract 1
DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for congenital anomalies of kidney and urinary tract 1 — screening already-approved drugs against its 6-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCongenital anomalies of kidney and urinary tract 1 maps to a 6-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for congenital anomalies of kidney and urinary tract 1 is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
deoxyribonuclease 1 (DNASE1) — DNASE1 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet 2-hydroxy-ethyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4AWN · 1.95 Å · ligand 2-[BIS-(2-HYDROXY-ETHYL)-AMINO]-2-HYDROXYMETHYL-PROPANE-1,3-DIOL (BTB). Experimental structure, not a prediction.
What the evidence adds up to
Congenital anomalies of the kidney and urinary tract are extremely common and are often diagnosed prenatally. Abnormalities may result in hydronephrosis, whether unilateral or bilateral, or in small dysplastic kidneys. The array of abnormalities is related to the embryologic stage at the time of the developmental insult, and these conditions range from insignificant to incompatible with life. CAKUT are important causes of chronic kidney disease in childhood. Most occur in isolation without a definite identifiable genetic defect, though familial occurrence has been reported rarely. The burden of CKD to a family in a developing country is enormous, and if more than one child is afflicted the situation is almost catastrophic.
A 2014 review describes lesions as varied as ureteral duplications, ureteropelvic junction obstruction, horseshoe kidney, posterior urethral valve, prune belly syndrome, obstructive renal dysplasia, nonmotile ciliopathies, and several syndromes associated with renal malformations including Meckel-Joubert, Bardet-Biedl, Zellweger, trisomies, VACTER-L, Potter, and sirenomelia, as well as autosomal dominant and autosomal recessive polycystic kidney disease. A 2020 study of perinatal autopsies analysed the prevalence of congenital urinary system anomalies, the correlation between antenatal ultrasound and postmortem findings, chromosomal test results, and associated extrarenal congenital malformations. A 2024 case report describes a 46-year-old woman with left acute pyelonephritis and bilateral duplicated ureters, noting that duplicated ureters can cause complications such as vesicoureteral reflux and ureteral obstruction; treatment involved antibiotics and outpatient urology follow-up.
The purpose of the 2014 review is not only to describe the congenital renal anomalies but also to analyse more recent therapeutic interventions that may modify the natural history of some of these severe conditions. Understanding the implications of common congenital urinary anomalies is key to detecting associated anomalies, initiating therapy, and avoiding both complications and unnecessary intervention. It is important to accurately diagnose the underlying cause of the renal and urinary tract anomaly to provide timely intervention and preservation of renal function.
What is still missing are large-scale prospective studies that stratify patients by specific anatomical defect and genetic background, funding for long-term follow-up of prenatal interventions, and controlled trials of surgical versus conservative management for the many subtypes of CAKUT where evidence remains anecdotal.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Fetal and Pediatric Pathology · 2014 · 174 citations · open access
Congenital Anomalies of the Kidney and the Urinary Tract (CAKUT)
AbstractThis article reviews the majority of Congenital Anomalies of the Kidney and Urinary Tract (CAKUT) with emphasis in Pediatric Pathology describing and illustrating lesions as varied as ureteral duplications, ureteropelvic junction obstruction, horseshoe kidney, posterior urethral valve and prune belly syndrome, obstructive renal dysplasia, nonmotile ciliopathies and several syndromes associated with renal malformations (Meckel-Joubert, short rib, Bardet-Biedl, asplenia/polysplenia, hereditary renal adysplasia, Zellweger, trisomies, VACTER-L, Potter, caudal dysplasia, and sirenomelia), as well as ADPK, and ARPK. The purpose of this review is not only to describe the congenital renal anomalies, but also to analyze the more recent therapeutic interventions that may modify the natural history of some of these severe conditions.
Developmental pathology of congenital kidney and urinary tract anomalies
AbstractCongenital anomalies of the kidneys or lower urinary tract (CAKUT) are the most common causes of renal failure in children and account for 25% of end-stage renal disease in adults. The spectrum of anomalies includes renal agenesis; hypoplasia; dysplasia; supernumerary, ectopic or fused kidneys; duplication; ureteropelvic junction obstruction; primary megaureter or ureterovesical junction obstruction; vesicoureteral reflux; ureterocele; and posterior urethral valves. CAKUT originates from developmental defects and can occur in isolation or as part of other syndromes. In recent decades, along with better understanding of the pathological features of the human congenital urinary tract defects, researchers using animal models have provided valuable insights into the pathogenesis of these diseases. However, the genetic causes and etiology of many CAKUT cases remain unknown, presenting challenges in finding effective treatment. Here we provide an overview of the critical steps of normal development of the urinary system, followed by a description of the pathological features of major types of CAKUT with respect to developmental mechanisms of their etiology.
American Journal of Roentgenology · 2012 · 17 citations
Genitourinary Imaging: Part 1, Congenital Urinary Anomalies and Their Management
AbstractOBJECTIVE: Congenital urinary anomalies may be symptomatic or encountered during imaging for other clinical indications. The array of abnormalities is related to the embryologic stage at the time of the developmental insult, and these abnormalities result in a spectrum of conditions ranging from insignificant to incompatible with life. CONCLUSION: Understanding the implications of common congenital urinary anomalies is the key to detecting associated anomalies, initiating therapy, and avoiding both complications and unnecessary intervention.
Congenital Anomalies of the Kidney and Urinary Tract
AbstractCongenital anomalies of the kidney and urinary tract are extremely common and are often diagnosed prenatally. Abnormalities may result in hydronephrosis, whether unilateral or bilateral, or in small dysplatic kidneys. It is important to accurately diagnose the underlying cause of the renal and urinary tract anomaly to provide timely intervention and preservation of renal function.
Indian Journal of Nephrology · 2013 · 5 citations · open access
Congenital anomalies of kidney and urinary tract in siblings: An uncommon condition
AbstractCongenital anomalies of kidney and urinary tract (CAKUT) are important causes of chronic kidney disease (CKD) in childhood. Most do not have a definite identifiable genetic defect and occur in isolation. Rarely, familial occurrence of CAKUT has been reported. The burden of CKD to a family in a developing country is enormous, and if more than one child is afflicted with the condition, the situation is almost catastrophic. We present here two families with siblings having upper and lower urinary tract obstruction.
Kafkas Journal of Medical Sciences · 2020 · 1 citations · open access
Urinary System Malformations and Associated Extrarenal Anomalies in Perinatal Autopsies
AbstractAim: Pediatric developmental and genetic renal anomalies are a known cause of mortality in the perinatal/neonatal period. These anomalies are associated with a wide range of extrarenal congenital malformations that influence the outcome of patients. This study aims to analyze the prevalence of congenital urinary system anomalies, the correlation between antenatal ultrasound findings and postmortem autopsy findings, chromosomal test results, the spectrum of developmental and genetic renal lesions, and associated congenital malformations.
Revista de la Facultad de Medicina Humana · 2024 · 0 citations · open access
Acute Left Pyelonephritis in Patient with Bilateral Low Back Pain and Ureteral Duplicity: Case Report
AbstractThe case presents a 46-year-old woman with left acute pyelonephritis and bilateral duplicated ureters. Acute pyelonephritis is a severe urinary tract infection that can vary in clinical presentation, from mild symptoms to sepsis. Lower back pain is a common symptom and can be unilateral or bilateral. Duplicated ureters are a congenital anomaly resulting in the transport of urine from the same kidney to the bladder, and can cause complications such as vesicoureteral reflux and ureteral obstruction. Treatment involved antibiotics and outpatient urology follow-up. The case underscores the importance of a meticulous diagnostic approach and consideration of underlying congenital anomalies that can complicate clinical management and treatment. The integration of clinical findings and complementary tests was essential for an accurate diagnosis and appropriate treatment plan.
AbstractAbnormalities of the urinary tract account for approximately 20% of all fetal malformations. Most of renal abnormalities are not life-threatening, but severe bilateral renal abnormalities account for 10% of all terminations for lethal fetal malformations. Abnormalities of the renal parenchyma can be divided into two main categories: anomalies of the renal echogenicity and cystic changes of the renal parenchyma. In this article, we present a review of the literature regarding the detection and management of fetal echogenic kidneys. Whenever fetal urinary tract malformations are detected, extra-urinary anomalies should be ruled out by a complete assessment of the fetus. The management of prenatally urinary tract malformations is moving towards a multidisciplinary approach with obstetricians, paediatric nephrologists and urologists.
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
