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CRISPR yields new potential “bubble boy” gene therapy

External Link - Source: SCOPE

About 1 in 50,000 baby boys are born with no immune cells — they have no way to molecularly protect themselves. The disease, called X-linked severe combined immunodeficiency, or SCID-X1, is more commonly known as the “bubble boy” disease because if babies born with SCID-X1 are not secluded in a hygienic “bubble,” they could contract an infection and die.

Now, through the gene-editing technology CRISPR-Cas9, Stanford scientist Matthew Porteus, MD, PhD, professor of pediatrics, is leading the charge for a new type of treatment that harnesses the particular class of stem cells that gives rise to immune and blood cells. In people with SCID-X1 these cells are robbed of their immune cell-generating abilities due to a mutation in a single gene called ILR2 gamma.

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