Mutations in antiquitin in individuals with pyridoxine-dependent seizures.

Mills, Philippa B; Struys, Eduard; Jakobs, Cornelis; Plecko, Barbara; Baxter, Peter; Baumgartner, Matthias; Willemsen, Michèl A A P; Omran, Heymut et al. · Nat Med · 2006

basic_science · Level V

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Abstract

We show here that children with pyridoxine-dependent seizures (PDS) have mutations in the ALDH7A1 gene, which encodes antiquitin; these mutations abolish the activity of antiquitin as a delta1-piperideine-6-carboxylate (P6C)-alpha-aminoadipic semialdehyde (alpha-AASA) dehydrogenase. The accumulating P6C inactivates pyridoxal 5'-phosphate (PLP) by forming a Knoevenagel condensation product. Measurement of urinary alpha-AASA provides a simple way of confirming the diagnosis of PDS and ALDH7A1 gene analysis provides a means for prenatal diagnosis.

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