DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hearing loss, autosomal recessive — screening already-approved drugs against its 24-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHearing loss, autosomal recessive maps to a 24-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 hearing loss, autosomal recessive 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
G protein signaling modulator 2 (GPSM2) — GPSM2 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 pgedrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4WND · 1.5 Å · ligand TRIETHYLENE GLYCOL (PGE). Experimental structure, not a prediction.
What the evidence adds up to
Fanconi anaemia, an autosomal recessive disorder, was found in a retrospective review of eight children to include asymmetrical bilateral conductive hearing loss more severe at lower frequencies, sometimes progressive. Four of the eight patients (50%) had hearing loss, and two (25%) had unilateral type I microtia and stenotic external ear canal. The authors recommended routine screening of all FA patients to recognise mild loss and prevent progression through rehabilitation.
A 2021 review outlines a strategy for developing genetic medicines for hearing loss, using the otoferlin gene (OTOF) as an exemplar. OTOF-mediated hearing loss is a sensorineural form caused by autosomal recessive mutations of the OTOF gene. The review states that delivering the correct version of the gene to a specific target cell within the inner ear has the potential to restore cochlear function for high-acuity physiologic hearing. A 2020 review notes that 119 nonsyndromic genes have been associated with hearing loss, and that gene therapy is emerging as a possible future management option. A 2025 collection of 31 papers highlights gene therapy applications for hearing loss alongside basic mechanisms and clinical studies.
A 2004 clinical genetic study of 144 patients with nonsyndromic hearing loss examined sex distribution, type, degree, symmetry, laterality, progression, aetiology, and inheritance pattern. A 1995 review presents a pipeline from family identification through linkage analysis, gene sequencing, and disease mechanism identification to gene-based therapy, noting that each mutation sits at a different stage of that pipeline. A 2024 case report describes bilateral sensorineural hearing loss after two days of oral fluoroquinolone therapy, a rare complication the authors recommend educating patients about.
What is still missing is large-scale, well-funded clinical trials that can move any of these gene therapy strategies from proof-of-concept in animal models or small case series into reliable treatments for defined patient subgroups. Patient stratification by specific gene mutation, age at intervention, and type of hearing loss remains largely unaddressed in the published work. The pipeline described in 1995 still has few mutations that have reached the gene-based therapy stage for hearing loss.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Acta Oto-Laryngologica · 2008 · 10 citations
Audiologic abnormalities of Fanconi anaemia
AbstractCONCLUSIONS: The most common audiologic manifestation in Fanconi anaemia (FA) was asymmetrical bilateral conductive hearing loss that was more severe at lower frequencies and in some cases had a progressive character. The routine screening of all patients diagnosed with FA allowed the recognition of mild hearing loss and the prevention of the deleterious effects of its progression with adequate rehabilitation measures. OBJECTIVES: FA is an autosomal recessive disease characterized by bone marrow failure, multiple congenital anomalies and increased susceptibility to malignancy. Otologic manifestations in FA include morphologic anomalies affecting the ear structures and hearing loss. This report is a retrospective review of the most important features, including audiologic features, in children with FA attending a paediatric hospital. SUBJECTS AND METHODS: The medical records of eight patients with FA were reviewed and patient demographics, physical abnormalities, haematological characteristics at diagnosis and otological and audiological features were analysed. RESULTS: Eight patients (five females, three males), aged between 3 and 13 years old, have been followed for at least 1 year in our hospital. In all, 50% (four of eight) of our population has hearing loss. It is an asymmetrical, bilateral, conductive hearing loss that is more severe at lower frequencies. Two patients (25%) have unilateral type I microtia and stenotic external ear canal.
Genetics of pediatric hearing loss: A functional perspective
AbstractOBJECTIVES: This article reviews the current role of genetics in pediatric hearing loss (HL). METHODS: A review of the current literature regarding the genetic basis of HL in children was performed. RESULTS: To date, 119 nonsyndromic genes have been associated with HL. There are also hundreds of syndromic causes that have HL as part of the clinical phenotype. CONCLUSIONS: Identifying HL genes coupled with clinical characteristics ("genotype-phenotype") yields a more accurate diagnosis and prognosis. Although the complexity of the auditory apparatus presents challenges, gene therapy is emerging and may be a viable management option in the future.
Journal of the American Academy of Audiology · 2021 · 4 citations
Genetic Medicine for Hearing Loss: <i>OTOF</i> as Exemplar
AbstractAbstract Millions of people worldwide have disabling hearing loss because one of their genes generates an incorrect version of some specific protein the ear requires for hearing. In many of these cases, delivering the correct version of the gene to a specific target cell within the inner ear has the potential to restore cochlear function to enable high-acuity physiologic hearing. Purpose: In this review, we outline our strategy for the development of genetic medicines with the potential to treat hearing loss. We will use the example of otoferlin gene ( OTOF )-mediated hearing loss, a sensorineural hearing loss due to autosomal recessive mutations of the OTOF gene.
Current Opinion in Otolaryngology & Head & Neck Surgery · 1995 · 2 citations
Modern clinical methods in genetic hearing loss
AbstractClinical methods in cases of genetic hearing loss have been influenced recently by both methodologic advances and specific findings made in the many research laboratories devoted to human genetic investigation. For the clinician, reports from these laboratories seem to suggest significant breakthroughs accompanied by allusions to clinical applicability. This review presents a rationale for the analysis of the many reports related to genetic hearing loss and their attending clinical impact. This rationale is based on a simplified view of the laboratory effort as a pipeline. A disease (mutation) passes through this pipeline on its way from family identification, through linkage analysis (chromosome localization), to gene sequencing and disease mechanism identification, and finally to gene-based therapy. Each stage of the investigation process has its own impact on clinical methods, and each mutation is in a particular spot in the pipeline at present. The pipeline itself is presented in this paper, along with the nature of the clinical impact of each level of laboratory findings.
American Journal of Audiology · 2004 · 1 citations
Clinical Genetic Study of 144 Patients With Nonsyndromic Hearing Loss
AbstractHearing loss constitutes an important category of congenital defects that can be isolated or part of the phenotypic spectrum of several syndromes. A clinical genetic study was performed on a sample of 144 patients with nonsyndromic hearing loss, establishing the sex distribution, type, degree, symmetry, laterality, progression, etiology, and, when possible, inheritance pattern.
Advanced Science · 2025 · 1 citations · open access
Hearing Loss: From Basic to Clinical Science
AbstractHearing loss (HL) affects over 1.5 billion people globally, with genetic factors accounting for ≈50% of congenital cases. Therefore, HL has become a global health issue, driving extensive research from basic science to clinical applications. This Special Collection includes a total of 31 papers, among which 9 are review papers, 21 are research article papers, 1 is a perspective paper, that highlight the basic mechanisms and possible protection methods of HL, the application of gene therapy for treating HL, and the clinical study and application in HL.
Zenodo (CERN European Organization for Nuclear Research) · 2024 · 0 citations · open access
A Rare Case of Bilateral Sensorineural Hearing Loss with Oral Fluoroquinolone Use
AbstractWe present a case exposing the rare complication of Sensorineural Hearing Loss (SNHL) following oral fluroquinolone therapy. The patient developed SNHL following two days of oral therapy. Though this is a rare complication, we recommend understanding and fully educating patients on fluroquinolone side effects and concerning symptoms to be aware of while undergoing fluoroquinolone therapy. Additionally, it is equally as important for physicians should look to optimize side effect profiles for any medical intervention they recommend.
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.
