| 摘要 |
The invention relates to a safer and efficient recombinant gene transfer system for ribozyme mediated repair of a target RNAin non-dividing and dividing cells, based on the Visna lentivirus (VLV) comprising: (a) a first plasmid comprising a sequence encoding the VLV Gag, (b) a second plasmid as transfer plasmid comprising a ribozyme cassette th the catalytic domain of a self-splizing ribozyme (class I intron) and a sequence encoding for the accurate counterpart of the target RNA to be repaired, (c) a third plasmid comprising a sequence encoding VLV Pol, (d) a fourth plasmid comprising sequences encoding VLV Vif, Rev and Tat, (e) a fifth plasmid comprising a sequence encoding an envelope protein Env, which is not derived from VLV, wherein the plasmids a and c to e do not encode for other VLV proteins and do not contain a competent packaging signal. |
