The purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Autologous hematopoietic stem cells with MT-gp91 retroviral vector
Seoul National University Hospital
Seoul, South Korea
The incidence of adverse events through 1 year
Time frame: 1 year
RCR, insertional mutagenesis, immune response against normal gp91 protein
Time frame: 1 year
Safety and efficacy of fludarabine/busulfan conditioning
Time frame: 1 year
Functional reconstitution of respiratory burst
Time frame: 1 year
Presence of vector-positive cells
Time frame: 1 year
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