• “Bubble Boy Disease” Treated with Gene Therapy

    N Engl J Med. April 17, 2019.

    A child from the 1970s became the iconic “bubble boy” who lived in a plastic enclosure to avoid germs due to his immunodeficiency disorder. Work done over the decades has recently played out with the successful use of gene therapy in eight children to treat what is known as “X-linked severe combined immunodeficiency disorder” or X-SCID. Here an innocuous virus was used to insert a missing gene (IL2RG) into bone marrow cells. This heralded the use of a safer virus than one used some 2 decades ago which had activated a cancer gene.

    It appears that this gene therapy in conjunction with targeted chemotherapy may offer hope to those with X-SCID, but as with any new intervention, only time will tell if there are inadvertent and unpredictable downstream consequences.

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