LAY TITLE: The development of stem cell therapy inside the womb to treat babies with muscular dystrophy

The aim of this project is to develop stem cell therapy inside the womb to treat muscular dystrophy before birth. The applicants have recently identified a new type of fetal stem cell which spreads to fetal organs to develop into new tissue, such as muscle.

The study will use a model system to test the ability of these stem cells, when given inside the womb, to spread to muscle tissue and prevent the effects of dystrophin deficiency which is one of the causes of muscular dystrophy. They will also investigate ways of increasing the number of new muscle cells formed, and ways of genetically-modifying stem cells so that they could eventually be used to correct dystrophin deficiency.

Demonstrating that fetal stem cells repair muscle damage inside the womb is an essential prerequisite before this type of treatment could be tried in affected pregnancies. If successful, it may open the door to the development of a new approach, not just to muscular dystrophy, but also to other disabling genetic conditions affecting bones, the brain and other vital organs.

27 Sussex Place
Regent's Park
London NW1 4SP

Tel: 020 7772 6400
Fax: 020 7724 7725
wellbeingofwomen@rcog.org.uk

Registered Charity No: 239281

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