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MOST parents dream of a 5-week-old baby who sleeps through the night, but Aga Warnell knew something was wrong. Her baby, Nina, just wasn't hungry in the way her other daughters had been.
Within weeks, Nina became very ill, says her father, Graeme. She was admitted to hospital with a rotavirus infection. Then she picked up pneumonia.
It turned out Nina had a condition called severe combined immunodeficiency (SCID). She had been born without an immune system due to a genetic defect. It is also known as "bubble boy" disease, since people affected have to live in a sterile environment. "The doctors said 'you need to prepare yourself for the fact that Nina probably isn't going to survive'," says Graeme.
A year-and-a-half later, Nina is a happy little girl with a functioning immune system. She has gene therapy – and its latest improvements – to thank for it.
SCID was the first condition to be treated with gene therapy more than 20 years ago. A virus was used to replace a faulty gene with a healthy one. But in subsequent...
Within weeks, Nina became very ill, says her father, Graeme. She was admitted to hospital with a rotavirus infection. Then she picked up pneumonia.
It turned out Nina had a condition called severe combined immunodeficiency (SCID). She had been born without an immune system due to a genetic defect. It is also known as "bubble boy" disease, since people affected have to live in a sterile environment. "The doctors said 'you need to prepare yourself for the fact that Nina probably isn't going to survive'," says Graeme.
A year-and-a-half later, Nina is a happy little girl with a functioning immune system. She has gene therapy – and its latest improvements – to thank for it.
SCID was the first condition to be treated with gene therapy more than 20 years ago. A virus was used to replace a faulty gene with a healthy one. But in subsequent...