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Mendeliome v1.4071 PFAS Zornitza Stark Marked gene: PFAS as ready
Mendeliome v1.4071 PFAS Zornitza Stark Gene: pfas has been classified as Amber List (Moderate Evidence).
Mendeliome v1.4071 PFAS Zornitza Stark Classified gene: PFAS as Amber List (moderate evidence)
Mendeliome v1.4071 PFAS Zornitza Stark Gene: pfas has been classified as Amber List (Moderate Evidence).
Mendeliome v1.4070 PFAS Zornitza Stark gene: PFAS was added
gene: PFAS was added to Mendeliome. Sources: Literature
Mode of inheritance for gene: PFAS was set to BIALLELIC, autosomal or pseudoautosomal
Publications for gene: PFAS were set to 40421664
Phenotypes for gene: PFAS were set to Inborn error of metabolism, MONDO:0019052, PFAS-related
Review for gene: PFAS was set to AMBER
Added comment: PMID 40421664 reports 2 individuals from 2 unrelated families with biallelic missense variants presenting with prematurity, short stature, seizures, and mild neurodevelopmental impairment. Functional studies in patient fibroblasts show ~30% PFAS protein, ~16% enzyme activity, impaired purinosome formation, and rescue of purinosome formation and FGAR levels by wild‑type PFAS, supporting pathogenicity.
Sources: Literature