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Intellectual disability v8.90 OPA1 Sarah Leigh Added comment: Comment on publications: PMID: 39233737 was identified by the Genomics England Applied Machine Learning (ML) team in a Biocuration-ML project for identifying new gene-disease associations using Natural Language Processing (NLP) and Generative AI techniques
Intellectual disability v8.90 OPA1 Sarah Leigh Publications for gene: OPA1 were set to 39233737
Intellectual disability v8.89 OPA1 Sarah Leigh Classified gene: OPA1 as Amber List (moderate evidence)
Intellectual disability v8.89 OPA1 Sarah Leigh Gene: opa1 has been classified as Amber List (Moderate Evidence).
Intellectual disability v8.88 OPA1 Sarah Leigh gene: OPA1 was added
gene: OPA1 was added to Intellectual disability. Sources: Literature
Q1_25_ promote_green tags were added to gene: OPA1.
Mode of inheritance for gene: OPA1 was set to MONOALLELIC, autosomal or pseudoautosomal, imprinted status unknown
Publications for gene: OPA1 were set to 39233737
Phenotypes for gene: OPA1 were set to Optic atrophy plus syndrome, OMIM: 125250
Review for gene: OPA1 was set to GREEN
Added comment: Heterozygous OPA1 variants have been associated with Optic atrophy 1 (OMIM:165500) and Optic atrophy plus syndrome, OMIM: 125250. PMID: 39233737 reports two unrelated cases of OMIM: 125250 with a "prominent neurological phenotype highly resembling clinical and
neuroradiological features of Leigh-like syndrome", including hypotonia and psychomotor delay. Each child had a de novo novel heterozygous OPA1 variant (NM_ 015560.3, c.888T>A, p.Asp296Glu and c.802T>C, p.Tyr268His). The mitochondria in the fibroblasts from these cases appeared to be fragmented with a reduced ATP production compared to controls; additionally, the amount of mtDNA was reduced by about a half in comparison with controls. Complementary studies in yeast suggested that these variants are pathogenic with a possible dominant negative effect (PMID: 39233737).
Sources: Literature