| abstract |
The instant invention provides silencer expression cassettes for delivery of RNAi therapy. The silencer cassettes of the instant invention encode an RNAi agent and lack means for replication within a host cell, inverted terminal repeats and further lacking one or more selectable markers. Optionally, the silencer cassettes lack CpG dinucleotides and/or the RNAi agent is within a scaffold of a naturally occurring miRNA. The composition comprising the silencer cassettes of the instant invention, as well as methods of use and suitable systems are also provided. |