Key result
Retrovirus constructs can mediate stable expression of rp47phox protein in human hematopoietic cell lines and restore it within p47phox-deficient EBV-BCL from patients with CGD.
Retrovirus-mediated gene transfer can successfully restore p47phox protein expression in deficient cell lines from patients with chronic granulomatous disease.
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Supports retroviral gene therapy feasibility in CGD; leaves open clinical translation pending in vivo and patient studies.
Cobbs et al. (1992) studied Autosomal chronic granulomatous disease (CGD). Retrovirus mediated expression of rp47phox vs. Antisense retroviral constructs and uninduced cells was evaluated on Expression of rp47phox protein. Retrovirus constructs can mediate stable expression of rp47phox protein in human hematopoietic cell lines and restore it within p47phox-deficient EBV-BCL from patients with CGD.