Gene transfer to primary chronic granulomatous disease monocytes

Article

Thrasher, A., Casimir, C., Kinnon, C., Morgan, G., Segal, A. and Levinsky, R. 1995. Gene transfer to primary chronic granulomatous disease monocytes. Lancet. 346 (8967), pp. 92-93. https://doi.org/10.1016/S0140-6736(95)92116-8
TypeArticle
TitleGene transfer to primary chronic granulomatous disease monocytes
AuthorsThrasher, A., Casimir, C., Kinnon, C., Morgan, G., Segal, A. and Levinsky, R.
Abstract

For somatic gene therapy to become a realistic therapeutic strategy for chronic granulomatous disease (CGD), we have to be able to assign the molecular lesion to a specific component of the NADPH oxidase and to confirm that transfer of a functional copy of the corresponding defective gene will result in correction of the cellular defect. We used an adenovirus vector expressing p47phox to transduce monocytes from patients with CGD. We showed by nitroblue-tetrazolium staining that NADPH-oxidase activity was restored to these cells. This technique offers a rapid means for molecular diagnosis. In the short term, this approach may have therapeutic potential.

Research GroupMolecular Biology group
PublisherLancet Publishing Group
JournalLancet
ISSN0140-6736
Publication dates
Print1995
Publication process dates
Deposited02 Dec 2009
Output statusPublished
Digital Object Identifier (DOI)https://doi.org/10.1016/S0140-6736(95)92116-8
LanguageEnglish
Permalink -

https://repository.mdx.ac.uk/item/81z46

Log in to edit
  • 38
    total views
  • 0
    total downloads
  • 0
    views this month
  • 0
    downloads this month

Export as

Related outputs

Efficient generation of transgenic mice by lentivirus-mediated modification of spermatozoa
Chandrashekran, A., Sarkar, R., Thrasher, A., Fraser, S., Dibb, N., Casimir, C., Winston, R. and Readhead, C. 2014. Efficient generation of transgenic mice by lentivirus-mediated modification of spermatozoa. The FASEB Journal. 28 (2), pp. 569-576. https://doi.org/10.1096/fj.13-233999
Lentiviral vector transduction of spermatozoa as a tool for the study of early development
Chandrashekran, A., Isa, I., Dudhia, J., Thrasher, A., Dibb, N., Casimir, C., Readhead, C. and Winston, R. 2014. Lentiviral vector transduction of spermatozoa as a tool for the study of early development. FEBS Open Bio. 4 (1), pp. 266-275. https://doi.org/10.1016/j.fob.2014.02.008
Targeted gene delivery using lentiviruses displaying surface ligands
Casimir, C. 2013. Targeted gene delivery using lentiviruses displaying surface ligands. The Journal of Gene Medicine.
Lentiviral transduction of spermatozoa (VITSPER): a simple method for the generation of transgenic mice
Casimir, C. 2013. Lentiviral transduction of spermatozoa (VITSPER): a simple method for the generation of transgenic mice. Nature Biotechnology.
Dyskeratosis congenita and the DNA damage response
Kirwan, M., Beswick, R., Walne, A., Hossain, U., Casimir, C., Vulliamy, T. and Dokal, I. 2011. Dyskeratosis congenita and the DNA damage response. British Journal of Haematology. 153 (5), pp. 634-643. https://doi.org/10.1111/j.1365-2141.2011.08679.x
Structure and expression of a newt cardio-skeletal myosin gene. Implications for the C value paradox
Casimir, C., Gates, P., Ross-Macdonald, P., Jackson, J., Patient, R. and Brockes, J. 1988. Structure and expression of a newt cardio-skeletal myosin gene. Implications for the C value paradox. Journal of Molecular Biology. 202 (2), pp. 287-296.
Evidence for dedifferentiation and metaplasia in amphibian limb regeneration from inheritance of DNA methylation.
Casimir, C., Gates, P., Patient, R. and Brockes, J. 1988. Evidence for dedifferentiation and metaplasia in amphibian limb regeneration from inheritance of DNA methylation. Development (Cambridge, England). 104 (4), pp. 657-68.
Purification of the 47 kDa phosphoprotein associated with the NADPH oxidase of human neutrophils
Teahan, C., Totty, N., Casimir, C. and Segal, A. 1990. Purification of the 47 kDa phosphoprotein associated with the NADPH oxidase of human neutrophils. The Biochemical Journal. 267 (2), pp. 485-489.
Characterization of the 47-kilodalton autosomal chronic granulomatous disease protein: tissue-specific expression and transcriptional control by retinoic acid
Rodaway, A., Teahan, C., Casimir, C., Segal, A. and Bentley, D. 1990. Characterization of the 47-kilodalton autosomal chronic granulomatous disease protein: tissue-specific expression and transcriptional control by retinoic acid. Molecular and cellular biology. 10 (10), pp. 5388-5396.
The alpha subunit of cytochrome b-245 mapped to chromosome 16
Bu-Ghanim, H., Casimir, C., Povey, S. and Segal, A. 1990. The alpha subunit of cytochrome b-245 mapped to chromosome 16. Genomics. 8 (3), pp. 568-70.
Autosomal recessive chronic granulomatous disease caused by deletion at a dinucleotide repeat
Casimir, C., Bu-Ghanim, H., Rodaway, A., Bentley, D., Rowe, P. and Segal, A. 1991. Autosomal recessive chronic granulomatous disease caused by deletion at a dinucleotide repeat. Proceedings of the National Academy of Sciences of the United States of America. 88 (7), pp. 2753-2757.
Isolation of cDNA coding for an ubiquitous membrane protein deficient in high Na+, low K+ stomatocytic erythrocytes
Stewart, G., Hepworth-Jones, B., Keen, J., Dash, B., Argent, A. and Casimir, C. 1992. Isolation of cDNA coding for an ubiquitous membrane protein deficient in high Na+, low K+ stomatocytic erythrocytes. Blood. 79 (6), pp. 1593-1601.
Identification of the defective NADPH-oxidase component in chronic granulomatous disease: a study of 57 European families
Casimir, C., Chetty, M., Bohler, M., Garcia, R., Fischer, A., Griscelli, C., Johnson, B. and Segal, A. 1992. Identification of the defective NADPH-oxidase component in chronic granulomatous disease: a study of 57 European families. European Journal of Clinical Investigation. 22 (6), pp. 403-406. https://doi.org/10.1111/j.1365-2362.1992.tb01481.x
Restoration of superoxide generation to a chronic granulomatous disease-derived B-cell line by retrovirus mediated gene transfer
Thrasher, A., Chetty, M., Casimir, C. and Segal, A. 1992. Restoration of superoxide generation to a chronic granulomatous disease-derived B-cell line by retrovirus mediated gene transfer. Blood. 80 (5), pp. 1125-1129.
Chronic granulomatous disease: towards gene therapy.
Thrasher, A., Segal, A. and Casimir, C. 1993. Chronic granulomatous disease: towards gene therapy. Immunodeficiency. 4 (1-4), pp. 327-33.
Low NADPH oxidase activity in Epstein-Barr-virus-immortalized B-lymphocytes is due to a post-transcriptional block in expression of cytochrome b558
Chetty, M., Thrasher, A., Abo, A. and Casimir, C. 1995. Low NADPH oxidase activity in Epstein-Barr-virus-immortalized B-lymphocytes is due to a post-transcriptional block in expression of cytochrome b558. The Biochemical Journal. 306, pp. 141-145.
Functional reconstitution of the NADPH-oxidase by adeno-associated virus gene transfer
Thrasher, A., De Alwis, M., Casimir, C., Kinnon, C., Page, K., Lebkowski, J., Segal, A. and Levinsky, R. 1995. Functional reconstitution of the NADPH-oxidase by adeno-associated virus gene transfer. Blood. 86 (2), pp. 761-5.
Generation of recombinant adeno-associated virus (rAAV) from an adenoviral vector and functional reconstitution of the NADPH-oxidase
Thrasher, A., De Alwis, M., Casimir, C., Kinnon, C., Page, K., Lebkowski, J., Segal, A. and Levinsky, R. 1995. Generation of recombinant adeno-associated virus (rAAV) from an adenoviral vector and functional reconstitution of the NADPH-oxidase. Gene therapy. 2 (7), pp. 481-485.
Molecular analysis in three cases of X91: variant chronic granulomatous disease
Bu-Ghanim, H., Segal, A., Keep, N. and Casimir, C. 1995. Molecular analysis in three cases of X91: variant chronic granulomatous disease. Blood. 86 (9), pp. 3575-3582.
Mapping of human non-muscle type cofilin (CFL1) to chromosome 11q13 and muscle-type cofilin (CFL2) to chromosome 14
Gillett, G., Fox, M., Rowe, P., Casimir, C. and Povey, S. 1996. Mapping of human non-muscle type cofilin (CFL1) to chromosome 11q13 and muscle-type cofilin (CFL2) to chromosome 14. Annals of Human Genetics. 60 (Pt 3), pp. 201-211.
Enhanced retroviral transduction of 5-fluorouracil-resistant human bone marrow (stem) cells using a genetically modified packaging cell line
Povey, J., Weeratunge, N., Marden, C., Sehgal, A., Thrasher, A. and Casimir, C. 1998. Enhanced retroviral transduction of 5-fluorouracil-resistant human bone marrow (stem) cells using a genetically modified packaging cell line. Blood. 92 (11), pp. 4080-4089.
Retroviral transduction of quiescent haematopoietic cells using a packaging cell line expressing the membrane-bound form of stem cell factor
Sehgal, A., Weeratunge, N. and Casimir, C. 1999. Retroviral transduction of quiescent haematopoietic cells using a packaging cell line expressing the membrane-bound form of stem cell factor. Gene therapy. 6 (6), pp. 1084-1091.
Haemophilias: advances towards genetic engineering replacement therapy.
Emilien, G., Maloteaux, J., Penasse, C., Goodeve, A. and Casimir, C. 2000. Haemophilias: advances towards genetic engineering replacement therapy. Clinical and laboratory haematology. 22 (6), pp. 313-23. https://doi.org/10.1046/j.1365-2257.2000.00332.x
Differentiation-dependent up-regulation of p47(phox) gene transcription is associated with changes in PU.1 phosphorylation and increased binding affinity
Marden, C., Stefanidis, D., Cunninghame-Graham, D. and Casimir, C. 2003. Differentiation-dependent up-regulation of p47(phox) gene transcription is associated with changes in PU.1 phosphorylation and increased binding affinity. Biochemical and Biophysical Research Communications. 305 (1), pp. 193-202. https://doi.org/10.1016/S0006-291X(03)00727-7
A functional ISRE is required for myeloid transcription of the p47(phox) gene
Marden, C., Cunninghame-Graham, D., Thrasher, A. and Casimir, C. 2003. A functional ISRE is required for myeloid transcription of the p47(phox) gene. Biochimica et biophysica acta. 1630 (2-3), pp. 117-122. https://doi.org/10.1016/j.bbaexp.2003.09.005
Enhancer-deleted retroviral vectors restore high levels of superoxide generation in a mouse model of CGD.
Schwickerath, O., Brouns, G., Thrasher, A., Kinnon, C., Roes, J. and Casimir, C. 2004. Enhancer-deleted retroviral vectors restore high levels of superoxide generation in a mouse model of CGD. Journal of Gene Medicine. 6 (6), pp. 603-15.
Growth factor displayed on the surface of retroviral particles without manipulation of envelope proteins is biologically active and can enhance transduction.
Chandrashekran, A., Gordon, M., Darling, D., Farzaneh, F. and Casimir, C. 2004. Growth factor displayed on the surface of retroviral particles without manipulation of envelope proteins is biologically active and can enhance transduction. Journal of Gene Medicine. 6 (11), pp. 1189-96.
Targeted retroviral transduction of c-kit+ hematopoietic cells using novel ligand display technology
Chandrashekran, A., Gordon, M. and Casimir, C. 2004. Targeted retroviral transduction of c-kit+ hematopoietic cells using novel ligand display technology. Blood. 104 (9), pp. 2697-2703. https://doi.org/10.1182/blood-2003-10-3717
Circulating haematopoietic progenitors are differentially reduced amongst subtypes of dyskeratosis congenita
Kirwan, M., Vulliamy, T., Beswick, R., Walne, A., Casimir, C. and Dokal, I. 2008. Circulating haematopoietic progenitors are differentially reduced amongst subtypes of dyskeratosis congenita. British Journal of Haematology. 140 (6), pp. 719-22. https://doi.org/10.1111/j.1365-2141.2008.06991.x
Exogenous TERC alone can enhance proliferative potential, telomerase activity and telomere length in lymphocytes from dyskeratosis congenita patients
Kirwan, M., Beswick, R., Vulliamy, T., Nathwani, A., Walne, A., Casimir, C. and Dokal, I. 2009. Exogenous TERC alone can enhance proliferative potential, telomerase activity and telomere length in lymphocytes from dyskeratosis congenita patients. British Journal of Haematology. 144 (5), pp. 771-81. https://doi.org/10.1111/j.1365-2141.2008.07516.x
Molecular cloning and characterization of grancalcin, a novel EF-hand calcium-binding protein abundant in neutrophils and monocytes
Boyhan, A., Casimir, C., French, J., Teahan, C. and Segal, A. 1992. Molecular cloning and characterization of grancalcin, a novel EF-hand calcium-binding protein abundant in neutrophils and monocytes. The Journal of Biological Chemistry. 267 (5), pp. 2928-2933.
Post-transcriptional regulation of the chicken thymidine kinase gene
Groudine, M. and Casimir, C. 1984. Post-transcriptional regulation of the chicken thymidine kinase gene. Nucleic Acids Research. 12 (3), pp. 1427-46.