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Int J Mol Sci ; 24(18)2023 Sep 11.
Artigo em Inglês | MEDLINE | ID: mdl-37762234

RESUMO

The CRB1 gene plays a role in retinal development and its maintenance. When disrupted, it gives a range of phenotypes such as early-onset severe retinal dystrophy/Leber congenital amaurosis (EOSRD/LCA), retinitis pigmentosa (RP), cone-rod dystrophy (CORD) and macular dystrophy (MD). Studies in CRB1 retinopathies have shown thickening and coarse lamination of retinal layers resembling an immature retina. Its role in foveal development has not yet been described; however, this retrospective study is the first to report foveal hypoplasia (FH) presence in a CRB1-related retinopathy cohort. Patients with pathogenic biallelic CRB1 variants from Moorfields Eye Hospital, London, UK, were collected. Demographic, clinical data and SD-OCT analyses with FH structural grading were performed. A total of 15 (48%) patients had EOSRD/LCA, 11 (35%) MD, 3 (9%) CORD and 2 (6%) RP. FH was observed in 20 (65%; CI: 0.47-0.79) patients, all of whom were grade 1. A significant difference in BCVA between patients with FH and without was found (p = 0.014). BCVA continued to worsen over time in both groups (p < 0.001), irrespective of FH. This study reports FH in a CRB1 cohort, supporting the role of CRB1 in foveal development. FH was associated with poorer BCVA and abnormal retinal morphology. Nonetheless, its presence did not alter the disease progression.


Assuntos
Distrofias de Cones e Bastonetes , Anormalidades do Olho , Amaurose Congênita de Leber , Degeneração Macular , Distrofias Retinianas , Retinose Pigmentar , Humanos , Estudos Retrospectivos , Retina , Distrofias Retinianas/genética , Retinose Pigmentar/genética , Proteínas do Olho/genética , Proteínas de Membrana/genética , Proteínas do Tecido Nervoso/genética
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