Dicerna Presents Preclinical Data on Primary Hyperoxaluria Type 1 Drug | GenomeWeb

NEW YORK (GenomeWeb) – Dicerna Pharmaceuticals this week announced preclinical data showing that its investigational primary hyperoxaluria type 1 (PH1) therapy DCR-PH1 could silence expression of its target by as much as 97 percent in a mouse model of the disease.

PH1 is a rare, inherited autosomal-recessive condition characterized by the liver's inability to metabolize a precursor of oxalate due to disruption of an enzyme called alanine-glyoxylate aminotransferase 1 (AGT1). As a result, calcium oxalate builds up in renal tubules causing kidney stones and fibrosis.

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