L1 retrotransposon-mediated stable gene silencing

Nuo Yang, Lin Zhang, Haig H. Kazazian

Research output: Contribution to journalArticlepeer-review

17 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1-8
Number of pages8
JournalNucleic acids research
Issue number6
StatePublished - 2005
Externally publishedYes

ASJC Scopus subject areas

  • Genetics


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