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.

journal_name

Placenta

journal_title

Placenta

authors

Coutelle C,Themis M,Waddington S,Gregory L,Nivsarkar M,Buckley S,Cook T,Rodeck C,Peebles D,David A

doi

10.1016/s0143-4004(03)00140-1

keywords:

subject

Has Abstract

pub_date

2003-10-01 00:00:00

pages

S114-21

eissn

0143-4004

issn

1532-3102

pii

S0143400403001401

journal_volume

24 Suppl B

pub_type

杂志文章,评审

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