Gene therapy for baby born without immune system

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Nina Warnell (above) was born with Severe Combined Immunodeficiency Syndrome, and is currently under going gene therapy treatment.

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.

Read the full article here: Baby born without an immune system given world-first gene therapy in the hope it will ‘re-boot’ her body’s defences

Additional Resources:

  • 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.
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