Therapeutic targeting of HYPDH/PRODH2 with N-propargylglycine offers a Hyperoxaluria treatment opportunity

Joanna Bons,Ada Tadeo,Gary K. Scott, Fadzai Teramayi,John J. Tanner,Birgit Schilling,Christopher C. Benz,Lisa M. Ellerby

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE（2024）

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摘要

N-propargylglycine prevents 4-hydroxyproline catabolism in mouse liver and kidney. N-propargylglycine is a novel suicide inhibitor of PRODH2 and induces mitochondrial degradation of PRODH2. PRODH2 is selectively expressed in liver and kidney and contributes to primary hyperoxaluria (PH). Preclinical evaluation of N-propargylglycine efficacy as a new PH therapeutic is warranted.

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关键词

4-hydroxyproline dehydrogenase (HYPDH/,PRODH2),N-propargylglycine(N-PPG),4-hydroxyproline (Hyp),Primary hyperoxaluria (PH)

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