title: Ex vivo gene therapy for HIV-1 treatment.
abstract: Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5(-/-) donor, even after discontinuation of conventional therapy, has energized the field. We review the status of current approaches as well as future directions in the areas of therapeutic targets, combinatorial strategies, vector design, introduction of therapeutics into stem cells and enrichment/expansion of gene-modified cells. Finally, we discuss recent advances towards clinical application of HIV-1 GT.
authors.affiliation: Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA.
authors.forename: Lisa J
authors.initials: LJ
authors.lastname: Scherer
authors.forename: John J
authors.initials: JJ
authors.lastname: Rossi
mesh_terms: D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D015658:HIV Infections / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D015497:HIV-1 / Q000235:genetics / Q000276:immunology; D006801:Humans; D019713:Receptors, CCR5 / Q000235:genetics / Q000276:immunology
chemical_list: D019713:Receptors, CCR5