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IEMbase 0318: CYP51A1-related lanosterol demethylase deficiency

Scope

Field Value
IEMbase ID 318
Nosology 14.7.19.01
Gene CYP51A1
External IDs OMIM:601637
Generated mapping UNMAPPED
Candidate DisMech targets Fuzzy candidate COA3-Related_COX_Deficiency.yaml rejected
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents this as autosomal recessive CYP51A1-related lanosterol demethylase deficiency. Alternate labels include CYP51 deficiency and cytochrome P450 family 51 deficiency.

Characteristic rows include cataract, erythema, hypopigmentation, self injury, and short stature. Additional clinical rows include developmental delay and microcephaly. No biochemical or treatment rows are present in the cached record.

DisMech phenotype coverage

The generated fuzzy candidate is COA3-Related_COX_Deficiency.yaml, but this is a lexical false positive. The local COA3 file covers COA3-related mitochondrial complex IV deficiency with peripheral neuropathy, exercise intolerance, obesity, short stature, and supportive metabolic care. It does not share the CYP51A1 gene, lanosterol demethylase mechanism, or sterol pathway scope.

There is no valid local CYP51A1 lanosterol demethylase deficiency target.

Concordance and completeness

Judgement: true local disease gap.

The current local candidate has only a nonspecific short-stature overlap and should not be used. IEMbase provides the initial review prompt set for future curation: cataract, erythema, hypopigmentation, self-injury, short stature, developmental delay, and microcephaly.

Curation actions

  • Do not map this record to COA3-related COX deficiency.
  • Add a standalone CYP51A1 lanosterol demethylase deficiency target if this sterol-biosynthesis disease is prioritized.
  • Look for source-backed biochemical sterol markers before modeling the entry, since the cached record has no biochemical rows.