Body cell gene therapy in toddlers

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To date germ line therapy has been carried out on animal eggs, sperm and embryos but at present it is illegal to do this on humans.

It is however possible to modify the faulty genes in the cells of a grown child or an adult to cure diseases like cystic fibrosis. This is called body cell gene therapy. Gene therapists have recently succeeded in curing a toddler of a life-threatening genetic condition that prevented him from developing an immune system. The toddler had a condition called severe combined immunodeficiency (SCID), which is caused by a single faulty gene.

Cells grown in the laboratory

Up until the body cell gene therapy treatment in March 2001, the 18-month-old boy lived in a sterile 'bubble' to prevent him picking up infections. Doctors took some of his bone marrow and used a non-infectious virus to carry a healthy version of the gene into the immune cells of his bone marrow.

The 'new improved' bone marrow was then re-implanted into the toddler where it gradually produced new immune cells. These immune cells entered the toddler's blood stream and protected him from infection. Now, thanks to gene therapy he is free to run around and his immune system is similar to that of other children his age.