EDITORIAL: "AUTOLOGOUS STEM CELL GENE THERAPY: TOWARD A UNIVERSAL PLATFORM FOR PERSONALIZED THERAPY"

Abstract

Intensive investigations are aimed at moving DNA sequencing technologies into the individual human genome sequencing era, thereby paving the way to tantalizing personalized medicine (see: Leamon et al., 2007 http://www.genome.gov/ 12513210; http://www.nih.gov/news/pr/aug2005/nhgri-08.htm). The development of routine patient genome sequencing will be instrumental in the wide use and optimization of emerging personalized stem cell gene therapy that primarily relies on gene targeting. Gene targeting is driven by homologous recombination and mediates DNA exchanges between chromosomal and transfecting/transducing DNA, thereby providing the means to modify at will target chromosomal DNA sequences (see: Capecchi, 1989). Gene targeting stands thus as the ultimate process to tackle inherited diseases since mutated sequences can be corrected and wild-type genomic homeostasis ideally restored (Bertolotti, 1996, 2000a and 2000b). Such a gene repair approach eliminates dysregulation and oncogenic hazards that hamper randomintegration of therapeutic DNA into host chromosomes (Bertolotti, 1998). However, unlike current clinical gene therapy that relies on random-integration of a single transgene to tackle most dysfunctions of a disease gene, gene targeting necessitates