n Retrovirus n Lentivirus (pictured) n Other viral vectors
1990 First approved gene therapy trial. Immune cells from 4-year-old
1992 ADA-SCID is successfully treated through gene therapy on stem cells harvested from bone marrow.
2000 Announcement that two boys in
2002 French SCID trial suspended after four children develop leukaemia as a result of the retrovirus vector.
2003 Chinese company Shenzhen SiBiono GeneTech gains approval for treating head and neck cancer with Gendicine, a modified adenovirus carrying a tumour-suppressor gene.
2003 Researchers in the US begin the first human trial using a modified lentivirus. It is a disabled HIV virus carrying a gene to inhibit replication. Trial is a success.
2009 Progression of the degenerative disease adrenoleukodystrophy is halted in two boys using gene therapy.
2010 An adult with blood disorder beta-thalassaemia no longer needs monthly blood transfusions following gene therapy to insert a corrected beta-globin gene into stem cells that make blood.
2011 Six people with clotting disorder haemophilia B see a reduction in symptoms after gene therapy on liver cells.
2012 Glybera becomes the first gene therapy drug to be approved in the West, with European approval to treat lipoprotein lipase deficiency.
2013 Two papers describe the treatment of children with a degenerative disorder called metachromatic leukodystrophy and immune disorder Wiskott-Aldrich syndrome using gene therapy (Science, doi.org/pnv; doi.org/ppk).
This week Announcement that five children with ADA-SCID have been successfully treated and are doing well.
Nina: born with a bad gene
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 trials, four young patients were diagnosed with leukaemia two years after receiving a similar treatment. An 18-year-old also died following a reaction to a virus used in gene therapy for a liver condition. It was the start of a rocky road (see "Trials and tribulations of gene therapy", right).
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