CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts

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CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts

Cataracts are the leading cause of vision loss in the world, although surgical treatment can restore vision in cataract patients. Until now, there have been no adequate animal models for in vivo studies of artificial lens safety and drug interactions. Genetic studies have demonstrated that GJA8 is involved in maintaining lens opacity and proper lens development. In this study, a cataract model ...

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Identification of a Novel GJA8 (Cx50) Point Mutation Causes Human Dominant Congenital Cataracts

Hereditary cataracts are clinically and genetically heterogeneous lens diseases that cause a significant proportion of visual impairment and blindness in children. Human cataracts have been linked with mutations in two genes, GJA3 and GJA8, respectively. To identify the causative mutation in a family with hereditary cataracts, family members were screened for mutations by PCR for both genes. Se...

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CRISPR/Cas9-mediated Dax1 knockout in the monkey recapitulates human AHC-HH.

Mutations in the DAX1 locus cause X-linked adrenal hypoplasia congenita (AHC) and hypogonadotropic hypogonadism (HH), which manifest with primary adrenal insufficiency and incomplete or absent sexual maturation, respectively. The associated defects in spermatogenesis can range from spermatogenic arrest to Sertoli cell only syndrome. Conclusions from Dax1 knockout mouse models provide only limit...

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Targeted Genes Sequencing Identified a Novel 15 bp Deletion on GJA8 in a Chinese Family with Autosomal Dominant Congenital Cataracts

BACKGROUND Congenital cataract (CC) is the leading cause of visual impairment or blindness in children worldwide. Because of highly genetic and clinical heterogeneity, a molecular diagnosis of the lens disease remains a challenge. METHODS In this study, we tested a three-generation Chinese family with autosomal dominant CCs by targeted sequencing of 45 CC genes on next generation sequencing a...

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Strabismus and Congenital Cataracts

Purpose: To study the prevalence, characteristics, indications for and results of strabismus treatment secondary to congenital cataracts. Materials and methods: This was a retrospective study of 21 cases from 29 with congenital cataracts with strabismus (unilateral in 11 and bilateral in 10 cases). In 20 patients surgery on the cataracts was performed, with an intraocular lens inserted in 15 pa...

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ژورنال

عنوان ژورنال: Scientific Reports

سال: 2016

ISSN: 2045-2322

DOI: 10.1038/srep22024