Recent research has been successful in treating a rare genetic disorder called Artemis-deficient Severe Combined Immunodeficiency (SCID), which is more commonly known as 'bubble boy disease'. This is due to a 1970s documentary about a child with the condition who had to live in a sterile, plastic bubble due to the lack of a functioning immune system. In the trial, 10 infants with the condition were given a gene-replacement therapy which partially or fully restored their immune systems.
The gene-replacement therapy involved extracting stem cells from the bone marrow of the infants, inserting corrected genetic information into the cells, and infusing them back into the children through an IV. After 6 to 16 weeks, follow-up blood tests showed that all the children had produced T cells and B cells. The researchers plan to follow the children for longer to monitor any potential side effects, and to conduct trials with more children.
This advancement in treating Artemis-deficient SCID is significant, as it could potentially save the lives of many children who are born with this rare condition. It could provide hope for families who are affected by the condition, and could potentially provide a better quality of life for those affected.