| Literature DB >> 34837429 |
Benjamin Billiet1, Patrizia Amati-Bonneau2,3, Valérie Desquiret-Dumas2,3, Khadidja Guehlouz1, Dan Milea4, Philippe Gohier1, Guy Lenaers2, Delphine Mirebeau-Prunier2,3, Johan T den Dunnen5, Pascal Reynier2,3, Marc Ferré2.
Abstract
Pathogenic variants of the nuclear receptor subfamily 2 group F member 1 gene (NR2F1) are responsible for Bosch-Boonstra-Schaaf optic atrophy syndrome (BBSOAS), an autosomal dominant disorder characterized by optic atrophy associated with developmental delay and intellectual disability, but with a clinical presentation which appears to be multifaceted. We created the first public locus-specific database dedicated to NR2F1. All variants and clinical cases reported in the literature, as well as new unpublished cases, were integrated into the database using standard nomenclature to describe both molecular and phenotypic anomalies. We subsequently pursued a comprehensive approach based on computed representation and analysis suggesting a refinement of the BBSOAS clinical description with respect to neurological features and the inclusion of additional signs of hypotonia and feeding difficulties. This database is fully accessible for both clinician and molecular biologists and should prove useful in further refining the clinical synopsis of NR2F1 as new data is recorded.Entities:
Keywords: BBSOAS; Bosch-Boonstra-Schaaf optic atrophy syndrome; COUP transcription factor 1 protein; COUP-TF1; NR2F1; database; neurodegenerative disorders; nuclear receptor subfamily 2 group F member 1; ontology; optic atrophy
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Year: 2021 PMID: 34837429 DOI: 10.1002/humu.24305
Source DB: PubMed Journal: Hum Mutat ISSN: 1059-7794 Impact factor: 4.878