Severe Combined Immune Deficiency Syndrome:
SCID is a rare, inherited disorder that affects the development and function of white blood cells.
It's characterized by a deficiency of T and B cell development and function, which makes people with SCID highly susceptible to infections.
SCID is also known as the “bubble boy disease” or “boy in the bubble” syndrome.
This is due to the publicity surrounding David Vetter, a boy with X-linked SCID who lived in a germ-free plastic bubble for 12 years.
Management:
Gene therapy can be used to treat severe combined immunodeficiency (SCID) by correcting the root cause of the disease.
Gene therapy involves:
Inserting healthy human genes into a patient's stem cells.
Washing the virus from the cells.
Transplanting the corrected cells back into the body.
The corrected cells divide and proliferate, passing on the normal gene copies to all the blood cells.
Gene therapy has been successful in treating children with XSCID and ADA deficiency SCID.
However, some treatments and trials have been halted due to concerns over the safety of viral vectors.
Other treatments for SCID include:
Bone marrow transplantation,
Enzyme therapy,
Avoiding live vaccines,
Antimicrobial prophylaxis, and
Isolation to prevent catching an infection.
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