Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules

Abstract

The present application discloses a lentiviral transfer system which includes: (i) a self-inactivating transfer vector comprising: multiple gene units, wherein each gene unit includes a heterologous nucleic acid sequence operably linked to a regulatory nucleic acid sequence; and (ii) a helper construct which lacks a 5′ LTR, wherein the 5′ LTR has been replaced with a heterologous promoter, in which the helper construct further comprises: a lentiviral env nucleic acid sequence containing a deletion, wherein the deleted env nucleic acid sequence does not produce functional env protein; and a packaging signal contains a deletion, wherein the deleted packaging signal is nonfunctional.

What is claimed is: 
     
         1 . A method for treating a condition, comprising administering to a patient a self-inactivating vector, wherein the self-inactivating vector comprises:
 multiple gene units, wherein each gene unit comprises a heterologous nucleic acid sequence operably linked to a regulatory sequence; and   a mammalian insulator sequence and splice acceptor and donor cites, wherein the self-inactivating vector is free of wPRE (wood-chuck hepatitis virus post-transcriptional element).