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Molecular basis of maple syrup urine disease: novel mutations at the E1 alpha locus that impair E1(alpha 2 beta 2) assembly or decrease steady-state E1 alpha mRNA levels of branched-chain alpha-keto acid dehydrogenase complex.

We report the occurrence of three novel mutations in the E1 alpha (BCKDHA) locus of the branched-chain alpha-keto acid dehydrogenase (BCKAD) complex that cause maple syrup urine disease (MSUD). An 8-bp deletion in exon 7 is present in one allele of a compound-heterozygous patient (GM-649). A single...

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Main Authors: Chuang, J. L., Fisher, C. R., Cox, R. P., Chuang, D. T.
Formato: Artigo
Idioma:Inglês
Publicado em: 1994
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1918348/
https://ncbi.nlm.nih.gov/pubmed/8037208
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