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Transgene optimization significantly improves SIN vector titers, gp91Phox expression and reconstitution of superoxide production in X-CGD cells

MORENO-CARRANZA, B ; GENTSCH, M ; BRENNER, S ; GREZ, M ; STEIN, S ; SCHAMBACH, A ; SANTILLI, G ; RUDOLF, E ; RYSER, M. F ; HARIA, S ; THRASHER, A. J ; BAUM, C

Gene therapy, 2009-01, Vol.16 (1), p.111-118 [Periódico revisado por pares]

London: Nature Publishing Group

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Título:
Transgene optimization significantly improves SIN vector titers, gp91Phox expression and reconstitution of superoxide production in X-CGD cells
Autor: MORENO-CARRANZA, B ; GENTSCH, M ; BRENNER, S ; GREZ, M ; STEIN, S ; SCHAMBACH, A ; SANTILLI, G ; RUDOLF, E ; RYSER, M. F ; HARIA, S ; THRASHER, A. J ; BAUM, C
Assuntos: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Animals ; Applied cell therapy and gene therapy ; Biological and medical sciences ; Biotechnology ; Blood diseases ; Cell Line, Tumor ; Fundamental and applied biological sciences. Psychology ; Gene Expression ; Gene therapy ; Genetic disorders ; Genetic Therapy - methods ; Genetic Vectors - administration & dosage ; Genetic Vectors - genetics ; Granulomatous Disease, Chronic - metabolism ; Granulomatous Disease, Chronic - therapy ; Health. Pharmaceutical industry ; Hematopoietic Stem Cells - metabolism ; Hematopoietic Stem Cells - virology ; Humans ; Immunomagnetic Separation ; Industrial applications and implications. Economical aspects ; Medical sciences ; Membrane Glycoproteins - genetics ; Membrane Glycoproteins - metabolism ; Mice ; Mice, Knockout ; NADPH Oxidase 2 ; NADPH Oxidases - genetics ; NADPH Oxidases - metabolism ; Patient safety ; Retroviridae - genetics ; Reverse Transcriptase Polymerase Chain Reaction - methods ; Superoxides - analysis ; Transduction, Genetic - methods ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Transgenes ; Virus Inactivation
É parte de: Gene therapy, 2009-01, Vol.16 (1), p.111-118
Notas: ObjectType-Article-1
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ObjectType-Feature-2
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Descrição: Gene therapy has proven to be of potential value for the correction of inherited hematopoietic disorders. However, the occurrence of severe side effects in some of the clinical trials has questioned the safety of this approach and has hampered the use of long terminal repeat-driven vectors for the treatment of a large number of patients. The development of self-inactivating (SIN) vectors with reduced genotoxicity provides an alternative to the currently used vectors. Our initial attempts to use SIN vectors for the correction of a myeloid disorder, chronic granulomatous disease, failed due to low vector titers and poor transgene expression. The optimization of the transgene cDNA (gp91(phox)) resulted in substantially increased titers and transgene expression. Most notably, transgene optimization significantly improved expression of a second cistron located downstream of gp91(phox). Thus, optimization of the transgene sequence results in higher expression levels and increased therapeutic index allowing the use of low vector copy numbers per transduced cell and weaker internal promoters.
Editor: London: Nature Publishing Group
Idioma: Inglês

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Transgene optimization significantly improves SIN vector titers, gp91Phox expression and reconstitution of superoxide production in X-CGD cells

MORENO-CARRANZA, B ; GENTSCH, M ; BRENNER, S ; GREZ, M ; STEIN, S ; SCHAMBACH, A ; SANTILLI, G ; RUDOLF, E ; RYSER, M. F ; HARIA, S ; THRASHER, A. J ; BAUM, C

London: Nature Publishing Group

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