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Scientists uncover new path toward treating a rare but deadly neurologic condition

BOSTON - Studies with a popular laboratory model, the nematode Caenorhabditis elegans, have revealed a possible therapeutic avenue for a rare but deadly condition in which children are born without the ability to make molybdenum cofactor (Moco) on their own. Though little known, Moco is essential for life and so far it has proven extremely difficult to stably isolate this compound in the laboratory, preventing its use as a therapeutic supplement. This new work, which appears in Genes & Development, reveals that nematodes genetically engineered to be Moco-deficient can take up Moco that is attached to several types of proteins. This suggests that such protein-Moco complexes could be used as a treatment for Moco deficiency in people.

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