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Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficien…

X-linked Severe Combined Immunodeficiency (XSCID)

National Institute of Allergy and Infectious Diseases (NIAID)NCT01306019