Safe vectors for gene therapy

Drug – bio-affecting and body treating compositions – Whole live micro-organism – cell – or virus containing – Genetically modified micro-organism – cell – or virus

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424 932, A61K 4800

Patent

active

057414866

ABSTRACT:
The present invention provides retroviral vectors comprising direct repeats flanking a sequence that is desired to be deleted upon reverse transcription in a host cell. In a preferred embodiment the sequence that is desired to be deleted is the retroviral cis-acting encapsidation sequence (E) essential for virus production in helper cells. In gene therapy embodiments, the E sequence is deleted upon reverse transcription in target cells, thus preventing spread of retroviral vectors to non-target cells in the event of infection with replication competent viruses. The retroviral vectors of the present invention thus provide safe vectors for gene therapy.

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