Apr
18
2019

Early days yet, but most common form of ‘Bubble boy’ disease appears… cured.

In “Our Neural Chernobyl” Bruce Sterling suggested that HIV would prove exceptionally useful, once we broke it on the wheel; it could be used to deliver gene therapies efficiently, quickly, and while bypassing the cell’s normal defenses. According to the BBC, we’re now using it to do just that: “US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or “bubble boy” disease.” The scientists in question are a bit more oblique about it:

Gene therapy developed at St. Jude Children’s Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time. The results appear in the April 18 issue of the New England Journal of Medicine.

“These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1,” said first and corresponding author Ewelina Mamcarz, M.D., of the St. JudeDepartment of Bone Marrow Transplantation and Cellular Therapy.

St Jude Children’s Research Hospital

I can understand why the press release would hesitate over using the term ‘HIV’ in the same paragraph as ‘treating children.’ Or same press release. Or even same continent. Also, the journalists may have gotten that detail wrong. That’s often a safe bet, in fact. Still, if this process works, there are a lot of things we can use the delivery system for. Just as long as we avoid the sentient raccoons, hey?

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