Gene Therapy Used to Treat X-Linked SCID

A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID).  Mustang Bio has licensed the treatment from St. Jude Children’s Research Hospital.  X-SCID is also known as “bubble boy” syndrome in which there are mutations in genes that develop immune cells.  The treatment involves giving X-SCID patients busulfan (a low dose cancer drug) before reinfusing them with their own gene modified stem cells.

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