In Vivo CRISPR/Cas9 Inhibition of Hepatic LDH as Treatment of Primary Hyperoxaluria

Scientists demonstrated a liver-specific inhibition of lactate dehydrogenase (LDH) that resulted in a drastic reduction of LDH levels in the liver of primary hyperoxaluria 1 (PH1) and PH3 mice after a single dose delivery of AAV8 vectors expressing CRISPR/Cas9 system, resulting in a reduced urine oxalate levels and kidney damage without signs of toxicity.