Nanophthalmos 1

disease
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Also known as nanophthalmos-1NNO1

Summary

Nanophthalmos 1 (MONDO:0010836) is a disease with 1 cohort gene.

At a glance

  • Cohort genes: 1
  • ClinVar variants: 9

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical namenanophthalmos 1
Mondo IDMONDO:0010836
MeSHC563983
OMIM600165
UMLSC1838502
MedGen325037
GARD0018625
Is cancer (heuristic)no

Also known as: nanophthalmos 1 · nanophthalmos-1 · NNO1

Data availability: 9 ClinVar variants.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary diseasenanophthalmiananophthalmos 1

Related subtypes (3): nanophthalmos 2, nanophthalmos 3, nanophthalmos 4

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

9 retrieved; paginated sample, class counts are floors:

6 pathogenic, 1 likely pathogenic, 1 uncertain significance, 1 conflicting classifications of pathogenicity

ClinVarVariant (HGVS)GeneClassificationReview
3894587NM_001127392.3(MYRF):c.3376-1G>AMYRFPathogenicno assertion criteria provided
3894589MYRF, 1-BP DEL, 789C (rs769274302)MYRFPathogenicno assertion criteria provided
3894590MYRF, 1-BP DUP, 789C (rs769274302)MYRFPathogenicno assertion criteria provided
3894591MYRF, ARG478PROMYRFPathogenicno assertion criteria provided
3894592MYRF, ARG986TERMYRFPathogenicno assertion criteria provided
635185NM_001127392.3(MYRF):c.3361del (p.Arg1121fs)MYRFPathogenicno assertion criteria provided
4540456NM_001127392.3(MYRF):c.1048del (p.Ser350fs)MYRFLikely pathogeniccriteria provided, single submitter
996604NM_001127392.3(MYRF):c.2036T>C (p.Val679Ala)MYRFConflicting classifications of pathogenicitycriteria provided, conflicting classifications
4819798NM_001127392.3(MYRF):c.1116-28A>GMYRFUncertain significancecriteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 1 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
MYRFOrphanet:647811Cardiac-urogenital syndrome

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
MYRFHGNC:1181ENSG00000124920Q9Y2G1Myelin regulatory factorclinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
MYRFMyelin regulatory factorConstitutes a precursor of the transcription factor.

Protein-family classification

Druggable: 0 · Difficult: 1 · Unknown: 0 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Transcription factor18.3×0.121

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
MYRFTranscription factornop53-like_TF_DNA-bd_sf, NDT80_DNA-bd_dom, MYRF_C2

Expression context

Cohort genes with no expression data: 0.

1 cohort gene are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)1
unknown0

Top tissues across cohort

TissueCohort genes
C1 segment of cervical spinal cord1
inferior vagus X ganglion1
middle frontal gyrus1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
MYRF223ubiquitousmarkermiddle frontal gyrus, C1 segment of cervical spinal cord, inferior vagus X ganglion

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
MYRF979

Structural data

PDB: 1 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
MYRFQ9Y2G12

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 0. Enrichment computed across 1 evidence-associated genes (0 with Reactome annotation).

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
central nervous system myelin maintenance12808.7×0.002MYRF
central nervous system myelination1991.3×0.002MYRF
positive regulation of myelination1766.0×0.002MYRF
response to immobilization stress1732.7×0.002MYRF
positive regulation of oligodendrocyte differentiation1674.1×0.002MYRF
protein autoprocessing1648.1×0.002MYRF
oligodendrocyte development1601.9×0.002MYRF
response to cocaine1581.1×0.002MYRF
oligodendrocyte differentiation1421.3×0.003MYRF
positive regulation of DNA-templated transcription127.9×0.036MYRF

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 1

Druggability breadth: 0 of 1 evidence-associated genes (0%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

SymbolMoleculesMax phase
MYRF00

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Pharmacogenomics

Cohort genes with a PharmGKB record: 1; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)0
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug1MYRF

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
MYRF0

Clinical trials & evidence

Clinical trials

Clinical trials: 0.