… disorder of phagocytic cells 1,2 . We report the initial results of nine severely affected X-linked …
and progenitor cell-based lentiviral gene therapy following myeloablative conditioning in …

… + cells from patients with X-linked chronic granulomatous disease used in this study (Intramural
National Institute of Allergy and Infectious Disease supported, institutional review board …

… Chronic granulomatous disease (CGD) is a … the X-linked CYBB gene encoding for gp91
phox (NOX2), the redox center of the NADPH oxidase complex (XCGD). Current treatments of …

… vectors (LVs) has emerged as a promising therapeutic option for CGD patients. We …
expression in X-linked chronic granulomatous disease (XCGD) mice. We found persistence of gene-…

… Regulated transgene expression may improve the safety and efficacy of hematopoietic
stem cell (HSC) gene therapy. Clinical trials for X-linked chronic granulomatous disease (X-CGD) …

… lentiviral vectors encoding CYBB gene … of GT treatment for CGD. Taken together, our results
further support the clinical development of a gene therapy protocol for X-CGD using lentiviral …

… cell (HSC) gene therapy. This is the first-in-human study of lentiviral HSC gene therapy with
vector … This article highlights a promising alternative curative treatment of X-CGD without an …

… In this regard, the use of lentiviral vectors might be one choice, as the in vitro transduction …
after leukemia was found in the X-linked severe combined immunodeficiency trials, possibly …

… In contrast to other genetic diseases such as X-linked severe … Therefore, lentiviral vectors
indeed represent a promising … cell gene therapy of X-CGD and other hematopoietic diseases …

… hand, lentiviral vector (LV)-mediated gene therapy has been … in treating monogenic diseases
such as X-linked severe … a lentiviral vector for ex vivo gene therapy of X-linked chronic …