At just three months old, Nina was diagnosed with a rare immune disorder called ADA-SCID, which effectively meant that she was born without an immune system. As toxins began building up and killing cells that fight infection, she became critically ill.
Nina’s parents were distraught to hear that their new-born baby could die within weeks. At the time they were living in Poland, where they were told that her only hope was a bone marrow transplant.
They set out to learn everything they could about their daughter’s rare genetic condition. After studying medical journals, Nina’s parents tracked down a cutting-edge gene therapy treatment at Great Ormond Street Hospital (GOSH).
Nina’s dad, Graeme, says: “We were told there were two ways of curing Nina. Either a transplant, which was risky, or gene therapy, which was less proven.”
Nina’s parents decided to try the gene therapy being pioneered at GOSH and came over to the UK to join a clinical trial. Nina became the first girl and only the fourth patient to undergo the treatment.
Once she was well enough, doctors extracted stem cells from Nina’s bone marrow and used a specially adapted, harmless virus to fix her faulty DNA in a lab. The corrected stem cells were then put back into Nina’s body so she could develop a working immune system.
Thanks to this pioneering treatment, Nina is now a healthy and happy little girl. Graeme says: “Now she can play with other children, go to school and we don’t have to worry about people coming to visit who might pass something on. We’ll never forget the support and kindness we got at GOSH – they really held us together.”
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