A proposed role for CDO1 in central nervous system development: Three children with rare missense variants and a neurological phenotype.

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Tác giả: Caitlin Barnes, Amber Begtrup, Amy Crunk, Hong Cui, Amy E Dameron, Leandra Folk, Maria J Guillen Sacoto, Alexis Heidlebaugh, Jane Juusola, Rachel Lombardo, Bobbi McGivern, Sureni V Mullegama, Olivia L Redlich, Adi Reich, Heather Riordan, Leah Rowe, Matthew T Snyder, Shelley Towner

Ngôn ngữ: eng

Ký hiệu phân loại: 573.86 *Central nervous system

Thông tin xuất bản: United States : HGG advances , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 57233

CDO1 encodes a non-heme iron dioxygenase, which is involved in cysteine metabolism. While CDO1 has been proposed to be involved in multiple physiological processes, an association with congenital disease has yet to be well defined. This study presents detailed clinical and molecular information on three individuals with overlapping neurological features. All three individuals were found to have rare, conserved, de novo variants clustered in a conserved region of the CDO1 gene with no alternative genetic etiology identified. Features present in all three individuals included EEG abnormality or seizure, movement abnormalities, hypertonia, encephalopathy, severe microcephaly (-4SD below mean), growth failure, feeding difficulty, and abnormal brain morphology. Other common features included global developmental delay, sleep disturbance, contractures, cerebral palsy, hyperreflexia, hearing loss, and hypoxic respiratory failure. This study provides evidence supporting an association between de novo CDO1 missense variants and human neurological disease.
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