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 studiedMice, 9 months
What changedA single dose reduced gene messages by ≥50% for 6 months in mouse tissues beyond the liver
The catchMouse muscle strength was not reported alongside sustained gene silencing