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Lentiviral Gene The...
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Dahl, MariaLund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine
(author)
Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice
- Article/chapterEnglish2015
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LIBRIS-ID:oai:gup.ub.gu.se/218005
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https://gup.ub.gu.se/publication/218005URI
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https://doi.org/10.1038/mt.2015.16DOI
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https://lup.lub.lu.se/record/5145357URI
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Subject category:art swepub-publicationtype
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Gaucher disease is caused by an inherited deficiency of the enzyme glucosylceramidase. Due to the lack of a fully functional enzyme, there is progressive build-up of the lipid component glucosylceramide. Insufficient glucosylceramidase activity results in hepatosplenomegaly, cytopenias, and bone disease in patients. Gene therapy represents a future therapeutic option for patients unresponsive to enzyme replacement therapy and lacking a suitable bone marrow donor. By proof-of-principle experiments, we have previously demonstrated a reversal of symptoms in a murine disease model of type 1 Gaucher disease, using gammaretroviral vectors harboring strong viral promoters to drive glucosidase beta-acid (GBA) gene expression. To investigate whether safer vectors can correct the enzyme deficiency, we utilized self-inactivating lentiviral vectors (SIN LVs) with the GBA gene under the control of human phosphoglycerate kinase (PGK) and CD68 promoter, respectively. Here, we report prevention of, as well as reversal of, manifest disease symptoms after lentiviral gene transfer. Glucosylceramidase activity above levels required for clearance of glucosylceramide from tissues resulted in reversal of splenomegaly, reduced Gaucher cell infiltration and a restoration of hematological parameters. These findings support the use of SIN-LVs with cellular promoters in future clinical gene therapy protocols for type 1 Gaucher disease.
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Doyle, AlexanderLund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine(Swepub:lu)med-axd
(author)
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Olsson, KarinLund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine(Swepub:lu)molm-kol
(author)
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Månsson, Jan-Eric,1946Gothenburg University,Göteborgs universitet,Institutionen för biomedicin, avdelningen för klinisk kemi och transfusionsmedicin,Institute of Biomedicine, Department of Clinical Chemistry and Transfusion Medicine(Swepub:gu)xmanja
(author)
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Marques, A. R. A.
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Mirzaian, M.
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Aerts, J. M.
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Ehinger, MatsLund University,Lunds universitet,Tumörmikromiljö,Sektion I,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Tumor microenvironment,Section I,Department of Clinical Sciences, Lund,Faculty of Medicine(Swepub:lu)efor-meh
(author)
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Rothe, M.
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Modlich, U.
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Schambach, A.
(author)
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Karlsson, StefanLund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine(Swepub:lu)molm-ska
(author)
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Avdelningen för molekylärmedicin och genterapiInstitutionen för laboratoriemedicin
(creator_code:org_t)
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In:Molecular Therapy: Elsevier BV23:5, s. 835-8441525-00161525-0024
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Dahl, Maria
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Doyle, Alexander
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Olsson, Karin
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Månsson, Jan-Eri ...
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Marques, A. R. A ...
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Mirzaian, M.
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Aerts, J. M.
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Ehinger, Mats
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Rothe, M.
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Modlich, U.
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Schambach, A.
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Karlsson, Stefan
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University of Gothenburg
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