Intrastriatal Delivery of Integration-Deficient Lentiviral Vectors in a Rat Model of Parkinson's Disease.

Standard integration-proficient lentiviral vectors (IPLVs) are effective at much lower doses than other vector systems and have shown promise in several gene therapy approaches. Their main drawback is the potential risk of insertional mutagenesis. Novel biosafety-enhanced integration-deficient lentiviral vectors (IDLVs) offer a significant improvement and comparable transduction efficacy to their integrating counterparts in some central nervous system applications. We describe here methods for (1) production of IDLVs (and IPLVs), (2) IDLV/IPLV delivery into the striatum of a rat model of Parkinson's disease, and (3) postmortem brain processing.