GENETIC MODIFICATION OF THE HYDROXYACID OXIDASE 1 GENE FOR TREATMENT OF PRIMARY HYPEROXALURIA

摘要

Disclosed are engineered nucleases that bind and cleave a recognition sequence within a hydroxyacid oxidase 1 (HAO1 ) gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins or nucleic acids encoding engineered nucleases of the invention, and the use of such compositions for treatment of primary hyperoxaluria type I.