title: The hopes and fears of in utero gene therapy for genetic disease--a review.
abstract: Somatic gene delivery in utero is a novel approach to gene therapy for genetic disease. It is based on the concept that application of gene therapy vectors to the fetus in utero may prevent the development of early disease related tissue damage, may allow targeting of otherwise inaccessible organs, tissues and still expanding stem cell populations and may also provide postnatal tolerance against the therapeutic transgenic protein. This review outlines the hypothesis and scientific background of in utero gene therapy and addresses some of the frequently expressed concerns raised by this still experimental, potentially preventive gene therapy approach. We describe and discuss the choice of vectors, of animal models and routes of administration to the fetus. We address potential risk factors of prenatal gene therapy such as vector toxicity, inadvertent germ line modification, developmental aberration and oncogenesis as well as specific risks of this procedure for the fetus and mother and discuss their ethical implications.
authors.affiliation: Gene Therapy Research Group, Division of Biomedical Science, Imperial College London, Sir Alexander Fleming Building, London SW7 2AZ, UK. c.coutelle@imperial.ac.uk
authors.forename: C
authors.initials: C
authors.lastname: Coutelle
authors.forename: M
authors.initials: M
authors.lastname: Themis
authors.forename: S
authors.initials: S
authors.lastname: Waddington
authors.forename: L
authors.initials: L
authors.lastname: Gregory
authors.forename: M
authors.initials: M
authors.lastname: Nivsarkar
authors.forename: S
authors.initials: S
authors.lastname: Buckley
authors.forename: T
authors.initials: T
authors.lastname: Cook
authors.forename: C
authors.initials: C
authors.lastname: Rodeck
authors.forename: D
authors.initials: D
authors.lastname: Peebles
authors.forename: A
authors.initials: A
authors.lastname: David
mesh_terms: D000328:Adult; D000818:Animals; D005260:Female; D005315:Fetal Diseases / Q000628:therapy*; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D015316:Genetic Therapy; D006801:Humans; D011247:Pregnancy