Literature DB >> 15883261

New DGK gene mutations in the hepatocerebral form of mitochondrial DNA depletion syndrome.

Michelangelo Mancuso1, Silvio Ferraris, Jacklyn Pancrudo, Annette Feigenbaum, Julian Raiman, John Christodoulou, David R Thorburn, Salvatore DiMauro.   

Abstract

OBJECTIVE: To document novel homozygous mutations in the gene for deoxyguanosine kinase (DGK) in 3 children with mitochondrial DNA depletion.
DESIGN: Clinical features included liver failure, hypotonia, and nystagmus in 2 siblings, and liver cirrhosis, optic dysplasia, nystagmus, and microcephaly in the third patient. We sequenced the whole coding region of the DGK gene.
RESULTS: We identified 2 novel homozygous mutations, G352A and C269T, that lead to truncated proteins.
CONCLUSION: These data confirm that DGK mutations typically affect the liver and brain.

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Year:  2005        PMID: 15883261     DOI: 10.1001/archneur.62.5.745

Source DB:  PubMed          Journal:  Arch Neurol        ISSN: 0003-9942


  9 in total

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