Development of a Genetic Substrate Reduction Therapy for MPS III using Antisense Oligonucleotides as therapeutic agents

2026-01-132026-01-13http://hdl.handle.net/10400.18/10685RNA therapeutics,Rare Diseases,Antisense Oligonucleotides,Pre-clinical validation, Medical and health sciencesDevelopment of a Genetic Substrate Reduction Therapy for MPS III using Antisense Oligonucleotides as therapeutic agentsASOs2cureMPSIII