Widespread gene transfection into the central nervous system of primates

We attempted in vivo gene transfection into the central nervous system (CNS ) of non-human primates using the hemagglutinating virus of Japan (HVJ)-AVE liposome, a newly constructed anionic type liposome with a lipid compositi on similar to that of HIV envelopes and coated by the fusogenic envelope pr oteins of inactivated HVJ. HVJ-AVE liposomes containing the lacZ gene were applied intrathecally through the cisterna magna of Japanese macaques. Wide spread transgene expression was observed mainly in the neurons. The lacZ ge ne was highly expressed in the medial temporal lobe, brainstem, Purkinje ce lls of cerebellar vermis and upper cervical cord (29.0 to 59.4% of neurons) . Intrastriatal injection of an HVJ-AVE liposome-lacZ complex made a focal transfection around the injection sites up to 15 mm. We conclude that the i nfusion of HVJ-AVE liposomes into the cerebrospinal fluid (CSF) space is ap plicable for widespread gene delivery into the CNS of large animals.