DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for LEOPARD syndrome 2 — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleLEOPARD syndrome 2 maps to a 3-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 leopard syndrome 2 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
Raf-1 proto-oncogene, serine/threonine kinase (RAF1) — RAF1 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 agsdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9MMQ · 2.9 Å · ligand PHOSPHOTHIOPHOSPHORIC ACID-ADENYLATE ESTER (AGS). Experimental structure, not a prediction.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Archives of Disease in Childhood · 1971 · 29 citations · open access
`Little Leopard' Syndrome: Description of 3 Cases and Review of 24
Abstract<h3>Introduction</h3> There is increasing interest in documenting the performance of individual Endoscopists, for their own education and to ensure quality standards. What has been lacking hitherto is an infrastructure to facilitate collection and analyses of data to allow practitioners to easily compile a ‘report card’ of their own practice or benchmark themselves against their peers. The ERCP Quality Network is a web based tool started in the USA.1 <h3>Methods</h3> Anonymised key data points (indications, sedation/anaesthesia, therapies, successes and adverse events), on each case are uploaded through a web based interface onto a central server hosted by Olympus America. This analysis is limited to the 51 endoscopists based in USA and the 12 in the UK who have each entered more than 30 cases. The results were evaluated in aggregate. <h3>Results</h3> The table 1 shows some of the areas where practice differed between countries. Those Endoscopists reporting their data in Britain are clearly doing less complex procedures as judged by the accepted complexity grade2 with lower (but acceptable) technical success rates. The differences were maintained when the comparison was restricted to cases performed with conscious sedation. The proportion of complex ‘grade 3’ cases was significantly higher in USA. The other striking difference is in the use of anaesthesia, that is, 3% versus 62%. <h3>Conclusion</h3> These data do not purport to reflect average UK and US practice, since the participants are self-selected, and not necessarily representative. The UK data compares favourably with the BSG ERCP audit in which only 77% of trained endoscopists achieved a cannulation rate of greater than 80%.3 The collection of this level of data about ones own practice is likely to become mandatory in the near future. In the UK the driver for collection of quality data is likely to be revalidation. The ERCP Quality Network is available to use and is a practical tool for self monitoring of quality outcomes and benchmarking against peers.
Case Reports in Dermatological Medicine · 2023 · 2 citations · open access
LEOPARD Syndrome with a Sporadic PTPN11 Mutation in a Saudi Patient
AbstractLEOPARD syndrome (LS) is a rare autosomal dominant inherited or sporadic genetic disorder caused commonly by missense mutations in the protein-tyrosine phosphatase-nonreceptor type 11 (PTPN11) gene. Due to its rarity and a high chance of misdiagnosis, the epidemiological profile of LS is poorly established. To the best of our knowledge, this is the second report with a documented PTPN11 gene mutation in Saudi Arabia.
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.
