Chronic Granulomatous Disease Clinical Trial
Official title:
Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)
This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.
Chronic granulomatous disease (CGD) is a rare disorder caused by inherited defects in the
NADPH oxidase multienzyme complex. It is associated with severe and life-threatening
bacterial and fungal infections. Approximately two-thirds of all CGD cases result from
mutations within the X-linked gp91phox gene (CYBB), followed by the autosomal recessive forms
of CGD, with defects in the gene coding for p47phox (NCF1) accounting for 10-30% of all CGD
cases.
The primary objectives are to evaluate the safety of the advanced self-inactivating
lentiviral vector TYF-CYBB and TYF-NCF1, the ex-vivo gene transfer clinical protocol and the
efficacy of immune reconstitution in patients overcoming frequent infections present at the
time of treatment, assessment of vector integration sites, and finally the long-term
correction of immune dysfunctions.
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