Antisense oligonucleotides delivered to the amniotic cavity in utero modulate gene expression in the postnatal mouse.

ABSTRACT: Congenital diseases account for a large portion of pediatric illness. Prenatal screening and diagnosis permit early detection of many genetic diseases. Fetal therapeutic strategies to manage disease processes in utero represent a powerful new approach for clinical care. A safe and effective fetal pharmacotherapy designed to modulate gene expression ideally would avoid direct mechanical engagement of the fetus and present an external reservoir of drug. The amniotic cavity surrounding the fetus could serve as an ideal drug reservoir. Antisense oligonucleotides (ASOs) are an established tool for the therapeutic modulation of gene expression. We hypothesize that ASOs administered to the amniotic cavity will gain entry to the fetus and modulate gene expression. Here, we show that an ASO targeti

SUBMITTER: Depreux FF 

PROVIDER: S-EPMC5175366 | biostudies-literature | 2016 Nov

REPOSITORIES: biostudies-literature