Ontology highlight
ABSTRACT:
SUBMITTER: Alterman JF
PROVIDER: S-EPMC6879195 | biostudies-literature | 2019 Aug
REPOSITORIES: biostudies-literature
Alterman Julia F JF Godinho Bruno M D C BMDC Hassler Matthew R MR Ferguson Chantal M CM Echeverria Dimas D Sapp Ellen E Haraszti Reka A RA Coles Andrew H AH Conroy Faith F Miller Rachael R Roux Loic L Yan Paul P Knox Emily G EG Turanov Anton A AA King Robert M RM Gernoux Gwladys G Mueller Christian C Gray-Edwards Heather L HL Moser Richard P RP Bishop Nina C NC Jaber Samer M SM Gounis Matthew J MJ Sena-Esteves Miguel M Pai Athma A AA DiFiglia Marian M Aronin Neil N Khvorova Anastasia A
Nature biotechnology 20190802 8
Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA architecture, divalent siRNA (di-siRNA), that supports potent, sustained gene silencing in the central nervous system (CNS) of mice and nonhuman primates following a single injection into the cerebrospinal fluid. Di-siRNAs are composed of two fully chemically modified, phosphorothioate-containing siRNAs connected by a linker. In mice, di-siRNAs induce ...[more]