Seventeen-month-old Nina Warnell suffers from Severe Combined Immunodeficiency Syndrome (SCID). She has all the appearance of a healthy child but her body is unable to fight even the mildest germs — even a cough or sneeze could kill her. Nina is missing a gene which produces an enzyme vital to the production of a healthy immune system.
The doctors have harvested her bone marrow and have re-engineered it using a new type of ‘reprogrammed virus’ to splice the vital gene she is missing into her DNA profile. The re-engineered bone marrow has been re-inserted into her body and they hope a fully functioning immune system will develop.
- “Bubble baby gene therapy ‘worth the risk’,” BBC News
The Great Ormond Street Hospital has been using gene therapy to help so-called “bubble babies” for over two decades. The techniques have been largely successful, but the treatment has a small chance of causing leukemia.
- Severe Combined Immunodeficiency Boston Children’s Hospital
Read about the genetic disorder at this website from the Boston Children’s Hospital.