Expanding the Spectrum of Oculocutaneous Albinism: Does Isolated Foveal Hypoplasia Really Exist?

被引:8
作者
Rocca, Camilla [1 ]
Tiberi, Lucia [1 ,2 ]
Bargiacchi, Sara [2 ]
Palazzo, Viviana [2 ]
Landini, Samuela [2 ]
Marziali, Elisa [3 ]
Caputo, Roberto [3 ]
Tinelli, Francesca [4 ]
Marchi, Viviana [4 ]
Benedetto, Alessandro [4 ]
Pagliazzi, Angelica [2 ]
Bacci, Giacomo Maria [3 ]
机构
[1] Univ Florence, Dept Biomed Expt & Clin Sci Mario Serio, I-50121 Florence, Italy
[2] Meyer Univ Hosp, Med Genet Unit, I-50139 Florence, Italy
[3] Meyer Univ Florence, Childrens Hosp A, Pediat Ophthalmol Unit, I-50139 Florence, Italy
[4] IRCCS Stella Maris Fdn, Dept Dev Neurosci, I-56128 Pisa, Italy
关键词
foveal hypoplasia; OCA; TYR; misrouting; VEP; Trios-WES; hypomorphic allele; good BCVA; albinism; TYROSINASE GENE; MUTATION; VEP; GENOMICS; CHIASM; FLASH; OCA;
D O I
10.3390/ijms23147825
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Oculocutaneous albinism is an autosomal recessive disorder characterized by the presence of typical ocular features, such as foveal hypoplasia, iris translucency, hypopigmented fundus oculi and reduced pigmentation of skin and hair. Albino patients can show significant clinical variability; some individuals can present with only mild depigmentation and subtle ocular changes. Here, we provide a retrospective review of the standardized clinical charts of patients firstly addressed for evaluation of foveal hypoplasia and slightly subnormal visual acuity, whose diagnosis of albinism was achieved only after extensive phenotypic and genotypic characterization. Our report corroborates the pathogenicity of the two common TYR polymorphisms p.(Arg402Gln) and p.(Ser192Tyr) when both are located in trans with a pathogenic TYR variant and aims to expand the phenotypic spectrum of albinism in order to increase the detection rate of the albino phenotype. Our data also suggest that isolated foveal hypoplasia should be considered a clinical sign instead of a definitive diagnosis of an isolated clinical entity, and we recommend deep phenotypic and molecular characterization in such patients to achieve a proper diagnosis.
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页数:16
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