title: [Principles and stakes of gene therapy].
abstract: As the human genome sequencing expands and the molecular bases of diseases are better understood, the tools that allow gene packaging and transfer are available. Transferring a therapeutical gene becomes a reality and indeed more than 100 clinical protocols are currently exploring a variety of applications, from the inherited genetic diseases such as cystic fibrosis to the acquired ones such as cancer. Herein, we describe the different gene transfer strategies applied to animals and humans and we present the advantages and limitations of the currently available vectors which, today, represent the limiting factor for efficient gene therapy.
authors.affiliation: Laboratoire de thérapie génique, CHU Hötel-Dieu, Nantes.
authors.forename: P
authors.initials: P
authors.lastname: Moullier
authors.forename: N
authors.initials: N
authors.lastname: Ferry
authors.forename: J M
authors.initials: JM
authors.lastname: Heard
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans
vernacular_title: Principes et enjeux de la thérapie génique.