COMPOSITIONS AND METHODS FOR TREATING SUBJECTS HAVING A HETEROZYGOUS ALANINE-GLYOXYLATE AMINOTRANSFERASE GENE (AGXT) VARIANT

摘要

The present invention provides methods for treating subjects suffering from a kidney stone disease carrying a heterozygous AGXT gene variant, methods for identifying such subjects, and compositions comprising nucleic acid inhibitors, e.g., double stranded ribonucleic acid (dsRNA) agents or single stranded antisense polynucleotide agents targeting lactate dehydrogenase A (LDHA) and/or hydroxyacid oxidase (HAO1), for treating such subjects.