Molecular Therapy Nucleic Acids
RNA platform sustained gene silencing beyond the liver in mice
One dose reduced target gene messages by more than 90% in several mouse tissues, with at least 50% silencing lasting six months.One dose reduced target gene messages by more than 90% in several mouse tissues, with at least 50% silencing lasting six months.
Mouse studyInterventions
Who was studiedC57Bl/6J mice, 9-month study
What changedGene activity in heart, aorta, and fat fell by ≥50% for up to 6 months after LiCO treatment
The catchThe mouse durability experiment tracked Sod1 RNA, not sustained protein suppression