Literature DB >> 36124184

A novel mutation of RPGR in a Chinese family with X-linked retinitis pigmentosa.

Hui-Hui Sun1, Jing-Cong Zhao1, Su-Ling Yang1, Jin-Dou Shi2, Yun-Shuo Wei1, Jian-Cang Wang1, Feng Gu3, Lu Chen1.   

Abstract

AIM: To identify potential mutations and elucidate the clinical findings of male patients and female carriers of X-linked retinitis pigmentosa (XLRP) in a Chinese family.
METHODS: A four generation pedigree was collected that consisted of 20 individuals. Genomic DNA was extracted from peripheral blood, and then the target fragments were amplified by PCR and sequenced directly. In addition, all affected patients and female carriers underwent comprehensively ophthalmic evaluation.
RESULTS: A novel mutation c.2865G>A p.W955X in RPGR gene was identified of this family, including four affected individuals and eight carriers. All male patients, aging from 7 to 31y, tended to have more various, even potentially deleterious clinical features of RP. At the same time, individuals with heterozygous mutations (carriers) manifested a wide spectrum of clinical features. Herein, only two male patients and three female carriers manifested pathological myopia (PM). Among the female carriers, half of subjects who harbor poor visual acuity suffered esotropia or exotropia. Additionally, 16.7% and 66.7% of carriers had abnormal electroretinogram (ERG) and fundus, respectively.
CONCLUSION: In this study, a novel mutation of the RPGR gene is identified, which broadens the spectrum of RPGR mutations, and elaborates the relationship between genotype and phenotype. International Journal of Ophthalmology Press.

Entities:  

Keywords:  RPGR; X-linked retinitis pigmentosa; nonsense mutation; phenotype

Year:  2022        PMID: 36124184      PMCID: PMC9453402          DOI: 10.18240/ijo.2022.09.03

Source DB:  PubMed          Journal:  Int J Ophthalmol        ISSN: 2222-3959            Impact factor:   1.645


  30 in total

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9.  The Spectrum of Structural and Functional Abnormalities in Female Carriers of Pathogenic Variants in the RPGR Gene.

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10.  Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.

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