Article
Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.
Blood - 2 Mar 2023
Wong Ryan L, Sackey Sarah, Brown Devin, Senadheera Shantha, Masiuk Katelyn, Quintos Jason P, Colindres Nicole, Riggan Luke, Morgan Richard A, Malech Harry L, Hollis Roger P, Kohn Donald B
Abstract excerpt
X-linked chronic granulomatous disease (X-CGD) is a primary immunodeficiency caused by mutations in the CYBB gene, resulting in the inability of phagocytic cells to eliminate infections. To design a lentiviral vector (LV) capable of recapitulating the endogenous regulation and expression of CYBB, a bioinformatics-guided approach was used to elucidate the cognate enhancer elements regulating the native CYBB gene....
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