Novel self-inactivating (SIN) lentiviral vectors

摘要

Novel retroviral LTRs are disclosed for use in lentiviral gene therapy vectors. The R region of the LTR is derived from a lentivirus, but lacks all or a portion of the lentiviral TAR sequence to increase the safety of the gene therapy vector. The TAR sequence can be replaced by comparable sequences from the R region from a non-lentiviral retrovirus, thereby generating a hybrid lentiviral/non-lentiviral R region which lacks TAR. Also disclosed are gene therapy vectors including the LTRs and methods of using the vectors in lentiviral-based gene therapy.