Fetal gene therapy: opportunities and risks

Wagner, Anna M; Schoeberlein, Andreina; Surbek, Daniel (2009). Fetal gene therapy: opportunities and risks. Advanced drug delivery reviews, 61(10), pp. 813-21. Amsterdam: Elsevier 10.1016/j.addr.2009.04.011

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Advances in human prenatal medicine and molecular genetics have allowed the diagnosis of many genetic diseases early in gestation. In-utero transplantation of allogeneic hematopoietic stem cells (HSC) has been successfully used as a therapy in different animal models and recently also in human fetuses. Unfortunately, clinical success of this novel treatment is limited by the lack of donor cell engraftment in non-immunocompromised hosts and is thus restricted to diseases where the fetus is affected by severe immunodeficiency. Gene therapy using genetically modified autologous HSC circumvents allogeneic HLA barriers and constitutes one of the most promising new approaches to correct genetic deficits in the fetus. Recent developments of strategies to overcome failure of efficient transduction of quiescent hematopoietic cells include the use of new vector constructs and transduction protocols. These improvements open new perspectives for gene therapy in general and for prenatal gene transfer in particular. The fetus may be especially susceptible for successful gene therapy due to the immunologic naiveté of the immature hematopoietic system during gestation, precluding an immune reaction towards the transgene. Ethical issues, in particular those regarding treatment safety, must be taken into account before clinical trials with fetal gene therapy in human pregnancies can be initiated.

Item Type:

Journal Article (Further Contribution)

Division/Institute:

04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Gynaecology
04 Faculty of Medicine > Pre-clinic Human Medicine > BioMedical Research (DBMR) > Unit Childrens Hospital > Forschungsgruppe Pränatale Medizin

UniBE Contributor:

Wagner, Anna Margaretha; Schoeberlein, Andreina and Surbek, Daniel

Subjects:

500 Science
500 Science > 570 Life sciences; biology
600 Technology > 610 Medicine & health

ISSN:

0169-409X

Publisher:

Elsevier

Language:

English

Submitter:

Factscience Import

Date Deposited:

04 Oct 2013 15:12

Last Modified:

24 Oct 2019 18:09

Publisher DOI:

10.1016/j.addr.2009.04.011

PubMed ID:

19426772

Web of Science ID:

000268289900006

BORIS DOI:

10.7892/boris.31595

URI:

https://boris.unibe.ch/id/eprint/31595 (FactScience: 196214)

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