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Fetal therapies – (Stem cell transplantation; enzyme replacement therapy; in utero genetic therapies)

Sagar, Rachel; David, Anna L; (2024) Fetal therapies – (Stem cell transplantation; enzyme replacement therapy; in utero genetic therapies). Best Practice & Research Clinical Obstetrics & Gynaecology , 97 , Article 102542. 10.1016/j.bpobgyn.2024.102542. (In press). Green open access

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Abstract

Advances in ultrasound and prenatal diagnosis are leading an expansion in the options for parents whose fetus is identified with a congenital disease. Obstetric diseases such as pre-eclampsia and fetal growth restriction may also be amenable to intervention to improve maternal and neonatal outcomes. Advanced Medicinal Therapeutic Products such as stem cell, gene, enzyme and protein therapies are most commonly being investigated as the trajectory of treatment for severe genetic diseases moves toward earlier intervention. Theoretical benefits include prevention of in utero damage, smaller treatment doses compared to postnatal intervention, use of fetal circulatory shunts and induction of immune tolerance. New systematic terminology can capture adverse maternal and fetal adverse events to improve safe trial conduct. First-in-human clinical trials are now beginning to generate results with a focus on safety first and efficacy second. If successful, these trials will transform the care of fetuses with severe early-onset congenital disease.

Type: Article
Title: Fetal therapies – (Stem cell transplantation; enzyme replacement therapy; in utero genetic therapies)
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.bpobgyn.2024.102542
Publisher version: https://doi.org/10.1016/j.bpobgyn.2024.102542
Language: English
Additional information: © The Author(s), 2024. This is an Open Access article distributed under the terms of the Creative Commons Attribution Licence (CC BY 4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. https://creativecommons.org/licenses/by/4.0/
Keywords: Fetal gene therapy, Fetal stem cell transplantation, Fetal gene editing, Fetal ultrasound, Prenatal diagnosis, Prenatal therapy
UCL classification: UCL
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 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/10198198
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