| Literature DB >> 36190665 |
Misbahuddin M Rafeeq1, Muhammad Umair2,3, Muhammad Bilal4, Alaa Hamed Habib1, Ahmed Waqas5, Ziaullah M Sain6, Mohammad Zubair Alam7,8, Raja Hussain Ali9.
Abstract
Cerebellar ataxias (CAs) comprise a rare group of neurological disorders characterized by extensive phenotypic and genetic heterogeneity. In the last several years, our understanding of the CA etiology has increased significantly and resulted in the discoveries of numerous ataxia-associated genes. Herein, we describe a single affected individual from a consanguineous family segregating a recessive neurodevelopmental disorder. The proband showed features such as global developmental delay, cerebellar atrophy, hypotonia, speech issues, dystonia, and profound hearing impairment. Whole-exome sequencing and Sanger sequencing revealed a biallelic nonsense variant (c.496A > T; p.Lys166*) in the exon 5 of the PRDX3 gene that segregated perfectly within the family. This is the third report that associates the PRDX3 gene variant with cerebellar ataxia. In addition, associated hearing impairment further delineates the PRDX3 associated gene phenotypes.Entities:
Keywords: Cerebellar ataxia; Nonsense variant; Novel variant; PRDX3; Profound deafness; Whole-exome sequencing
Year: 2022 PMID: 36190665 DOI: 10.1007/s10048-022-00701-9
Source DB: PubMed Journal: Neurogenetics ISSN: 1364-6745 Impact factor: 3.017