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.
Read more at the source article fiercebiotech.com