Skip to content

IEMbase 0141: ATIC-related AICAR transformylase-IMP cyclohydrolase deficiency

Scope

Field Value
IEMbase ID 141
Nosology 16.2.21.01
Gene ATIC
External IDs OMIM:608688; OMIM:601731; ORPHA:250977
Generated mapping UNMAPPED
Candidate DisMech targets No valid ATIC/AICA-ribosiduria target found; generated catecholamine-synthesis candidate is false
Review date 2026-07-07

IEMbase phenotype signal

IEMbase represents this as ATIC-related AICAR transformylase-IMP cyclohydrolase deficiency, with alternate label AICAribosiduria and abbreviation AICAR. Treatability is marked unknown.

The characteristic biochemical row is markedly increased urinary AICA riboside. The enzyme-testing row records markedly decreased fibroblast AICAR transformylase/IMP cyclohydrolase activity. Clinical rows include high nasal bridge, low-set ears, thin upper lip, wide mouth, blindness, coarse facial features, dysmorphic features, and intellectual disability. No treatment rows are listed.

DisMech phenotype coverage

No local standalone ATIC/AICA-ribosiduria entry was found. The generated catecholamine-synthesis candidate is a false pathway/acronym neighbor and does not involve ATIC or de novo purine synthesis.

Adenylosuccinate_Lyase_Deficiency.yaml mentions ATIC/AICA-ribosiduria only in a reference title about purinosome assembly. That is pathway context, not a disease-level target for ATIC deficiency.

Concordance and completeness

Judgement: true unmapped local disease gap.

The IEMbase record points to ATIC deficiency with urinary AICA riboside accumulation, reduced bifunctional enzyme activity, dysmorphism, blindness, and intellectual disability. Current DisMech has adjacent purine-metabolism content for ADSL deficiency and Lesch-Nyhan syndrome, but not ATIC/AICA-ribosiduria.

Curation actions

  • Keep this record unmapped.
  • Reject the catecholamine-synthesis candidate and do not map to ADSL deficiency.
  • Future curation should add an ATIC/AICA-ribosiduria target with AICA riboside accumulation, reduced AICAR transformylase/IMP cyclohydrolase activity, intellectual disability, blindness, and craniofacial dysmorphism.