Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector.

Gene Therapy, 14 April 2011; doi:10.1038/gt.2011.45.

Gimenez-Cassina A, Wade-Martins R, Gomez-Sebastian S, Corona JC, Lim F, Diaz-Nido J.

Department of Molecular Neurobiology, Centro de Biología Molecular Severo Ochoa, Cantoblanco, Spain.

Keywords: FRDA, gene-based therapies, long-term persistent transgene expression, entire 135 kb FXN genomic DNA locus, bacterial artificial chromosome (iBAC), herpes simplex virus type 1 (HSV-1), -based vector.