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University College London, EGA – Institute for Women’s Health

The University College London (UCL) Elizabeth Garrett Anderson (EGA) Institute for Women’s Health brings together clinicians and researchers from a diverse range of disciplines to make a real and sustainable difference to women’s and babies health

In the Department of Maternal and Fetal Medicine, scientists and clinicians collaborate with a unifying aim to improve outcomes for pregnant women and their babies through improved diagnostic and treatment using cutting edge technologies and innovative treatments.

The team lead by Dr Pascale V Guillot develops the next generation of stem cell and gene therapies through personalised medicine to improve the quality of life of children suffering from bone fragility.

They use both primary mesenchymal stem cells isolated from the amniotic fluid, but also creates induced mesenchymal stem cells from induced pluripotent stem cells (iPSCs), themselves derived from skin fibroblast or urine epithelial cells.

The team also uses the factors released by such cells in tiny sacks called small extracellular vesicles, or exosomes. They modify the content of these exosomes to boost their efficacy and develop new therapeutics to counteract bone fragility and bone mass loss. This technology is currently applied to finding new treatments for skeletal dysplasia osteogenesis imperfecta.

Osteogenesis imperfecta is a rare genetic disease with prenatal onset affecting bones and connective tissue and characterised by fragile bones that break easily. It is caused by genetic defects that result in insufficient production or abnormal type I collagen, which is the main component of bones. They use a combination of iPSCs and gene-editing technology to develop personalised cell-based and cell-free therapeutics for the treatment of osteogenesis imperfecta.

Findings are applicable to other skeletal pathologies characterised by bone loss, as a result of prolonged immobilisation, hormonal changes (menopause), low gravity as it is the case for astronauts or diseases such as osteoporosis.