Gene therapy for lysosomal storage disorders.

Abstract

Lysosomal storage diseases (LSDs) are a group of single-gene disorders that have proven to be highly informative in revealing the merits of gene transfer as a technology platform. Over the past several years considerable progress has been made in delivering therapeutic genes to peripheral tissues as well as the central nervous system. The current leading vectors for direct genetic modification of target cells in vivo are derived from adeno-associated viruses (AAV) and lentiviruses. These vectors are capable of conferring widespread, robust, and sustained expression of a given gene in several mouse models of LSDs. Here we review recent progress using recombinant AAV and lentiviruses to treat various LSDs and the remaining challenges to translate the results in mice to human patients.