Knockdown of lactate dehydrogenase by adeno‐associated virus‐delivered CRISPR/Cas9 system alleviates primary hyperoxaluria type 1

Knockdown of lactate dehydrogenase by adeno‐associated virus‐delivered CRISPR/Cas9 system alleviates primary hyperoxaluria type 1

Abstract Background Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder caused by endogenous overproduction of hepatic oxalate, leading to hyperoxaluria, recurrent calcium oxalate kidney stones, and end‐stage renal disease. Lactate dehydrogenase (LDH) is an ideal target for diminishing oxa...

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Bibliographic Details
Main Authors: Rui Zheng, Xiaoliang Fang, Xi Chen, Yunteng Huang, Guofeng Xu, Lei He, Yueyan Li, Xuran Niu, Lei Yang, Liren Wang, Dali Li, Hongquan Geng
Format: Article
Language:English
Published: Wiley 2020-12-01
Series:Clinical and Translational Medicine
Subjects:
adeno‐associated virus
CRISPR/Cas9
gene therapy
lactate dehydrogenase
primary hyperoxaluria type 1
Online Access:https://doi.org/10.1002/ctm2.261

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