title: L1 retrotransposon-mediated stable gene silencing.
abstract: RNA interference (RNAi) is widely used for functional studies and has been proposed as a potential therapeutic agent. Current RNAi systems are largely efficient, but have limitations including transient effect, the need for viral handling and potential insertional mutations. Here, we describe a simple L1 retrotransposon-based system for the delivery of small interfering RNA (siRNA) and stable silencing in human cells. This system demonstrated long-term siRNA expression and significant reduction in both exogenous and endogenous gene expression by up to 90%. Further characterization indicated that retrotransposition occurred in a controlled manner such that essentially only one RNAi-cassette was integrated into the host genome and was sufficient for strong interference. Our system provides a novel strategy for stable gene silencing that is easy and efficient, and it may have potential applications for ex vivo and in vivo molecular therapy.
authors.affiliation: Department of Genetics, University of Pennsylvania Philadelphia, PA 19104, USA.
authors.forename: Nuo
authors.initials: N
authors.lastname: Yang
authors.forename: Lin
authors.initials: L
authors.lastname: Zhang
authors.forename: Haig H
authors.initials: HH
authors.lastname: Kazazian
mesh_terms: D005821:Genetic Techniques; D049452:Green Fluorescent Proteins / Q000235:genetics; D006367:HeLa Cells; D006801:Humans; D020084:Long Interspersed Nucleotide Elements; D016133:Polymerase Chain Reaction; D034622:RNA Interference
chemical_list: D049452:Green Fluorescent Proteins