¹Department of Experimental Medical Research, CNS Gene Therapy Unit, Section for Neuroscience, Wallenberg Neuroscience Center, Lund University, 22184 Lund, Sweden

Correspondence: Cecilia Lundberg, E-mail: johan.jakobsson@med.lu.se; , E-mail: cecilia.lundberg@med.lu.se

Received 12 September 2005; Revised 28 October 2005; Accepted 8 November 2005.

Abstract

Lentiviral vectors have been used extensively as gene transfer tools for the central nervous system throughout the past decade since they transduce most cell types in the brain, resulting in high-level and long-term transgene expression. This review discusses some of the recent progress in this field, including preclinical gene therapy experiments in disease models, development of regulated vectors, and the application of siRNA's using lentiviral vectors. We also describe some of the features that make lentiviral vectors a likely candidate for human gene therapy in the brain.