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Gene therapy for obstetric conditions

Spencer, RN; Carr, DJ; David, AL; (2014) Gene therapy for obstetric conditions. Fetal and Maternal Medicine Review , 25 (3-4) pp. 147-177. 10.1017/S0965539515000030. Green open access

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Abstract

The first clinical trials of gene therapy in the 1990s offered the promise of a new paradigm for the treatment of genetic diseases. Over the decades that followed the challenges and setbacks which gene therapy faced often overshadowed any successes. Despite this, recent years have seen cause for renewed optimism. In 2012 Glybera™, an adeno-associated viral vector expressing lipoprotein lipase, became the first gene therapy product to receive marketing authorisation in Europe, with a licence to treat familial lipoprotein lipase deficiency. This followed the earlier licensing in China of two gene therapies: Gendicine™ for head and neck squamous cell carcinoma and Oncorine™ for late-stage nasopharyngeal cancer. By this stage over 1800 clinical trials had been, or were being, conducted worldwide, and the therapeutic targets had expanded far beyond purely genetic disorders. So far no trials of gene therapy have been carried out in pregnancy, but an increasing understanding of the molecular mechanisms underlying obstetric diseases means that it is likely to have a role to play in the future. This review will discuss how gene therapy works, its potential application in obstetric conditions and the risks and limitations associated with its use in this setting. It will also address the ethical and regulatory issues that will be faced by any potential clinical trial of gene therapy during pregnancy.

Type: Article
Title: Gene therapy for obstetric conditions
Open access status: An open access version is available from UCL Discovery
DOI: 10.1017/S0965539515000030
Publisher version: http://dx.doi.org/10.1017/S0965539515000030
Language: English
Additional information: This article has been published in a revised form in Fetal and Maternal Medicine Review: http://dx.doi.org/10.1017/S0965539515000030. This version is free to view and download for private research and study only. Not for re-distribution, re-sale or use in derivative works. Copyright © Cambridge University Press 2015.
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL EGA Institute for Womens Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL EGA Institute for Womens Health > Maternal and Fetal Medicine
URI: https://discovery.ucl.ac.uk/id/eprint/1498777
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