Xue Ding

Pharmacological readthrough and base editing for ANK1 nonsense mutations in an erythroid model of hereditary spherocytosis

Findings provide exploratory proof-of-concept for targeted molecular intervention in ANK1 nonsense mutation-associated HS, highlighting TRIDs and ABEs as mechanistically distinct strategies with complementary strengths and limitations and suggesting their relative utility may depend on mutation-specific and clinical co...