Antisense Oligonucleotide–Mediated MSH3 Suppression Reduces Somatic CAG Repeat Expansion in Huntington’s Disease iPSC–Derived Striatal Neurons

Medicine, Neurology, Life Sciences, Cell Biology, Biochemistry, Genetics,
Huntington's disease   CAG repeat expansion   antisense oligonucleotide   iPSC-derived neurons   MSH3 protein  

Therapeutic Potential of ASO-Mediated MSH3 Suppression in Huntington’s Disease Academic Background Huntington’s disease (HD) is a neurodegenerative disorder caused by abnormal expansion of the CAG repeat sequence in the huntingtin gene (HTT). This expanded CAG repeat continues to expand somatically over time, driving the onset and progression of th...