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

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...

Construction of human 3D striato-nigral assembloids to recapitulate medium spiny neuronal projection defects in Huntington’s disease

Construction of human 3D striato-nigral assembloids to recapitulate medium spiny neuronal projection defects in Huntington’s disease

Constructing Human 3D Striatum-Substantia Nigra Organoids to Model Medium Spiny Neuron Projection Deficits in Huntington’s Disease Background Introduction Huntington’s Disease (HD) is a neurodegenerative disorder leading to significant deterioration of the motor system, primarily characterized by defects in medium spiny neurons (MSNs) that project ...