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Literature summary for 1.1.3.15 extracted from

  • Zabaleta, N.; Barberia, M.; Martin-Higueras, C.; Zapata-Linares, N.; Betancor, I.; Rodriguez, S.; Martinez-Turrillas, R.; Torella, L.; Vales, A.; Olaguee, C.; Vilas-Zornoza, A.; Castro-Labrador, L.; Lara-Astiaso, D.; Prosper, F.; Salido, E.; Gonzalez-Aseguinolaza, G.; Rodriguez-Madoz, J.R.
    CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I (2018), Nat. Commun., 9, 5454 .
    View publication on PubMedView publication on EuropePMC

Application

Application Comment Organism
medicine application of oan in vivo CRISPR/Cas9-mediated substrate reduction therapy to treat primary hyperoxaluria type I that results in excessive hepatic oxalate production causing end-stage renal disease. A single systemic administration of an AAV8-CRISPR/Cas9 vector targeting glycolate oxidase, prevents oxalate overproduction and kidney damage, with no signs of toxicity in Agxt1-/- mice Mus musculus

Organism

Organism UniProt Comment Textmining
Mus musculus Q9WU19
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Synonyms

Synonyms Comment Organism
HAO1
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Mus musculus