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Hematologic Disorders : X-linked Severe Combined Immunodeficiency (SCID-X1)
X-linked severe combined immunodeficiency (SCID-X1) is a terrible disease of childhood caused by changes in genes. Patients with this disease normally die in the first year of life if immune reconstitution is not achieved. Patients can be successfully treated by hematopoietic stem cell transplantation, particularly if they have an HLA matched sibling donor. Gene therapy using retroviral vectors to transduce autologous hematopoietic stem cells can be used for patients that lack a matched sibling donor and results in significant immune reconstitution in most patients.
However, 5 out of 20 patients have developed leukemia due to insertional mutagenesis in these initial gene therapy trials. We now propose to use a novel self-inactivating lentiviral vector that has been designed to reduce the potential for insertional mutagenesis. These vector supernatants will be used to treat newly diagnosed SCID-X1 patients who lack a matched sibling donor, who are less than 12 months of age but are older than 3.5 months of age.
Brian Sorrentino, MD
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105 USA
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