Exome sequencing in a family with intellectual disability, early onset spasticity, and cerebellar atrophy detects a novel mutation in EXOSC3

G Zanni, C Scotton, C Passarelli, M Fang, S Barresi… - Neurogenetics, 2013 - Springer
G Zanni, C Scotton, C Passarelli, M Fang, S Barresi, B Dallapiccola, B Wu, F Gualandi…
Neurogenetics, 2013Springer
Whole exome sequencing in two-generational kindred from Bangladesh with early onset
spasticity, mild intellectual disability, distal amyotrophy, and cerebellar atrophy transmitted
as an autosomal recessive trait identified the following two missense mutations in the
EXOSC3 gene: a novel p. V80F mutation and a known p. D132A change previously
associated with mild variants of pontocerebellar hypoplasia type 1. This study confirms the
involvement of RNA processing proteins in disorders with motor neuron and cerebellar …
Abstract
Whole exome sequencing in two-generational kindred from Bangladesh with early onset spasticity, mild intellectual disability, distal amyotrophy, and cerebellar atrophy transmitted as an autosomal recessive trait identified the following two missense mutations in the EXOSC3 gene: a novel p.V80F mutation and a known p.D132A change previously associated with mild variants of pontocerebellar hypoplasia type 1. This study confirms the involvement of RNA processing proteins in disorders with motor neuron and cerebellar degeneration overlapping with spinocerebellar ataxia 36 and rare forms of hereditary spastic paraplegia with cerebellar features.
Springer
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