Gene therapy has been talked about for quite some time, but recent news has turned a hope into a reality for babies born with adenosine deaminase (ADA)-deficient SCID, severe combined immunodeficiency.
Babies born with SCID are at high risk for infection and illness. Even simple ailments that would require most of us only some extra rest and cough medicine can lead to hospitalizations or even death. It’s difficult to diagnose due to it’s rrarityand it’s lack of obvious physical symptoms but simple everyday activities could prove incredibly dangerous for children with SCID.
The natural habits for babies to pick things up and put things in their mouths, the many different hands that may be picking up and holding them, playdates who have a cough, all my poses serious risk. For this reason, along with other immuneodeficiny conditions, SCID has been sometimes referred to as the “bubble baby” disease. Parents must often keep their child in a metaphorical bubble to avoid exposing their baby to germs and viruses.
There are precautions and treatments available for immunodeficiency patients…but no cure. Until, after three decades of dedication, researchers at UCLA successfully cured 18 babies, infants as young as 3 months up to four year olds, through gene therapy. These children reportedly now have fully functioning immune systems.
What is gene therapy?How was it done? The children’s own blood stem cells were genetically altered, to correct the genetic defect causing the deficiency. It was reported that the procedure left no side effects. This success seems nothing short of a miracle. Not only has it changed, and secured, the lives of 18 children and their families, this hurdle in medical care has given renewed hope to many other types of patients.
While this particular research targets only the genetic mutation causing adenosine deaminase deficient SCID, it has cemented the possibility that similar research could effectively cure previously chronic hereditary diseases. UCLA plans to begin moving forward with gene therapy research for sickle cell anemia.
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