Extending the critical regions for mutations in the non-coding gene RNU4ATAC in another patient with Roifman Syndrome

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Compound heterozygous mutations in the noncoding RNU4ATAC cause Roifman Syndrome by disrupting minor intron splicing

Roifman Syndrome is a rare congenital disorder characterized by growth retardation, cognitive delay, spondyloepiphyseal dysplasia and antibody deficiency. Here we utilize whole-genome sequencing of Roifman Syndrome patients to reveal compound heterozygous rare variants that disrupt highly conserved positions of the RNU4ATAC small nuclear RNA gene, a minor spliceosome component that is essential...

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ژورنال

عنوان ژورنال: Clinical Case Reports

سال: 2018

ISSN: 2050-0904

DOI: 10.1002/ccr3.1830