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boy treated with gene therapy

Scientists cure “bubble boy” disease with help of an improved gene therapy

Researchers declared that ten newborn children with a rare genetic disorder, the “bubble boy disease” were cured with the help of gene therapy.

With the help of this treatment, the babies have been cured of the disorder without any side effects or post-treatment complications. Scientists carrying out research hoped for this result for decades but had always received setbacks until now.

In 2003, researchers tried to use gene therapy for treating Severe Combined Immunodeficiency Disease, but they stopped midway as it was detected that the therapy gave them cancer. The present treatment does not come with any such dangerous side effects and scientists hope that it can be used for other rare diseases too such as sickle cell disease.

Children born with SCID did not have a properly working immune system and without receiving any treatment they did not even make it past their first birthday. Even simple illnesses such as common cold were fatal for these children. These children were kept in very protected environments and it gave rise to the name “bubble boy“. However, a boost in the mortality rate has been observed recently owing to the advanced detection tests and treatments such as bone marrow transplants. Unfortunately, even these treatments have complications and they make the patients dependent on regular dosages of immunoglobulin.

The latest gene therapy has been developed by St. Jude Children’s Research Hospital and UCSF Benioff Children’s Hospital based in San Francisco. The therapy rectifies the genetic defects which are there in the DNA of the babies just after they are born, which helps the body to develop the parts of the immune system that are missing.

After the extraction of blood stem cells from the bone marrow of the infants, researchers used a virus as a means of transport to send the corrected version of the defective gene to the stem cells of the patients. The rectified cells were reinfused into the body of the patients where the proliferation of the cells took place to grow healthy immune cells.

Scientists took special care in not enabling the genes which cause cancer, so they added “insulators” with the virus such that surrounding genes would not get affected when the virus is inserted into DNA. Apart from this, the patients were also given chemotherapy to a small extent for clearing the existing cells from the bone marrow so that proliferation of the corrected cells can occur in a better way.

It was an emotional day for the announcement at the St. Jude Children’s Research Hospital, as the team leader Brian Sorrentino had spent his last days fighting against his cancer to finish the work on the treatment.