In vivo CRISPR-Cas9 inhibition of hepatic LDH as treatment of primary hyperoxaluria
Rebeca Martínez-Turrillas, Ángel Martín-Mallo, Saray Rodríguez-Diaz, Natalia Zapata‐Linares, Paula Rodríguez-Márquez, Patxi San Martín‐Úriz, Amaia Vilas‐Zornoza, María Eréndira Calleja-Cervantes, Eduardo Salido, Felipe Prósper, Juan R. Rodríguez-Madoz
Abstract
genome editing using CRISPR-Cas9 systems would represent a valuable tool for improved therapeutic approaches for PH.
Topics & Concepts
CRISPRIn vivoCas9Genome editingPrimary hyperoxaluriaBiologyComputational biologyKidneyChemistryBiochemistryGeneGeneticsOrgan Donation and TransplantationCRISPR and Genetic EngineeringPrion Diseases and Protein Misfolding