Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression
Recombinant adenoviruses expressing human beta-glucuronidase (AxCAhGUS) and CTLA-4Ig (AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine model of mucopolysaccharidosis type VII (MPSVII). Seven days after the intravenous administration of AxCAhGUS, high levels of be...
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| Veröffentlicht in: | Molecular therapy 2000-05, Vol.1 (5 Pt 1), p.406-413 |
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| creator | Kosuga, M Takahashi, S Sasaki, K Li, X K Fujino, M Hamada, H Suzuki, S Yamada, M Matsuo, N Okuyama, T |
| description | Recombinant adenoviruses expressing human beta-glucuronidase (AxCAhGUS) and CTLA-4Ig (AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine model of mucopolysaccharidosis type VII (MPSVII). Seven days after the intravenous administration of AxCAhGUS, high levels of beta-glucuronidase (GUSB) activity were observed in the liver, spleen, heart, lung, kidney, and serum, while viral DNA was predominantly detected in the liver. To investigate the contribution of in vivo cross-correction of GUSB between the liver and other organs, we injected the serum obtained from the transduced mice into untreated MPSVII mice. Similar distributions of GUSB activity were observed in the serum-injected mice, suggesting that GUSB activities detected in the extrahepatic organs were due to the cross-correction rather than the direct gene transduction. This result also suggested that maintaining high levels of GUSB in the systemic circulation was essential for the effective treatment of MPSVII. To achieve this, we injected AxCAhGUS and AxCACTLA-4Ig into MPSVII mice. Serum GUSB activity was sustained at high levels for more than 200 days and morphological normalization of the liver and spleen was observed for a year. This suggests that long-term therapeutic efficacy in visceral organs of MPSVII is achievable by coexpression of CTLA-4Ig through an in vivo cross-correction pathway. |
| doi_str_mv | 10.1006/mthe.2000.0067 |
| format | Article |
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Seven days after the intravenous administration of AxCAhGUS, high levels of beta-glucuronidase (GUSB) activity were observed in the liver, spleen, heart, lung, kidney, and serum, while viral DNA was predominantly detected in the liver. To investigate the contribution of in vivo cross-correction of GUSB between the liver and other organs, we injected the serum obtained from the transduced mice into untreated MPSVII mice. Similar distributions of GUSB activity were observed in the serum-injected mice, suggesting that GUSB activities detected in the extrahepatic organs were due to the cross-correction rather than the direct gene transduction. This result also suggested that maintaining high levels of GUSB in the systemic circulation was essential for the effective treatment of MPSVII. To achieve this, we injected AxCAhGUS and AxCACTLA-4Ig into MPSVII mice. Serum GUSB activity was sustained at high levels for more than 200 days and morphological normalization of the liver and spleen was observed for a year. This suggests that long-term therapeutic efficacy in visceral organs of MPSVII is achievable by coexpression of CTLA-4Ig through an in vivo cross-correction pathway.</description><identifier>ISSN: 1525-0016</identifier><identifier>EISSN: 1525-0024</identifier><identifier>DOI: 10.1006/mthe.2000.0067</identifier><identifier>PMID: 10933961</identifier><language>eng</language><publisher>United States: Elsevier Limited</publisher><subject>Abatacept ; Adenoviridae - genetics ; Adenoviridae - metabolism ; Adenoviruses ; Animals ; Antigens, CD ; Antigens, Differentiation - genetics ; Antigens, Differentiation - metabolism ; beta-Galactosidase - biosynthesis ; Bone marrow ; Cells, Cultured ; Cloning ; CTLA-4 Antigen ; Disease ; DNA Primers - chemistry ; DNA, Viral - metabolism ; Enzymes ; Gene Expression ; Gene therapy ; Genetic Therapy ; Genetic Vectors ; Genotype ; Glucuronidase - genetics ; Glucuronidase - metabolism ; Humans ; Immunoconjugates ; Immunoglobulin Fc Fragments - metabolism ; Kidney - enzymology ; Kidney - pathology ; Liver ; Liver - enzymology ; Liver - pathology ; Lung - enzymology ; Lung - pathology ; Medical research ; Mice ; Mice, Inbred C57BL ; Mucopolysaccharidosis VII - genetics ; Mucopolysaccharidosis VII - metabolism ; Mucopolysaccharidosis VII - therapy ; Peptides ; Receptor, IGF Type 2 - metabolism ; Recombinant Fusion Proteins - genetics ; Recombinant Fusion Proteins - metabolism ; Research centers ; Spleen ; Spleen - enzymology ; Spleen - pathology</subject><ispartof>Molecular therapy, 2000-05, Vol.1 (5 Pt 1), p.406-413</ispartof><rights>Copyright Nature Publishing Group May 2000</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c2747-d51c2ae0d101f141b5372ecb80c2d499616f223294c9dee741e4327cc4bffba33</citedby><cites>FETCH-LOGICAL-c2747-d51c2ae0d101f141b5372ecb80c2d499616f223294c9dee741e4327cc4bffba33</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,776,780,27901,27902</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/10933961$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Kosuga, M</creatorcontrib><creatorcontrib>Takahashi, S</creatorcontrib><creatorcontrib>Sasaki, K</creatorcontrib><creatorcontrib>Li, X K</creatorcontrib><creatorcontrib>Fujino, M</creatorcontrib><creatorcontrib>Hamada, H</creatorcontrib><creatorcontrib>Suzuki, S</creatorcontrib><creatorcontrib>Yamada, M</creatorcontrib><creatorcontrib>Matsuo, N</creatorcontrib><creatorcontrib>Okuyama, T</creatorcontrib><title>Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression</title><title>Molecular therapy</title><addtitle>Mol Ther</addtitle><description>Recombinant adenoviruses expressing human beta-glucuronidase (AxCAhGUS) and CTLA-4Ig (AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine model of mucopolysaccharidosis type VII (MPSVII). Seven days after the intravenous administration of AxCAhGUS, high levels of beta-glucuronidase (GUSB) activity were observed in the liver, spleen, heart, lung, kidney, and serum, while viral DNA was predominantly detected in the liver. To investigate the contribution of in vivo cross-correction of GUSB between the liver and other organs, we injected the serum obtained from the transduced mice into untreated MPSVII mice. Similar distributions of GUSB activity were observed in the serum-injected mice, suggesting that GUSB activities detected in the extrahepatic organs were due to the cross-correction rather than the direct gene transduction. This result also suggested that maintaining high levels of GUSB in the systemic circulation was essential for the effective treatment of MPSVII. To achieve this, we injected AxCAhGUS and AxCACTLA-4Ig into MPSVII mice. Serum GUSB activity was sustained at high levels for more than 200 days and morphological normalization of the liver and spleen was observed for a year. This suggests that long-term therapeutic efficacy in visceral organs of MPSVII is achievable by coexpression of CTLA-4Ig through an in vivo cross-correction pathway.</description><subject>Abatacept</subject><subject>Adenoviridae - genetics</subject><subject>Adenoviridae - metabolism</subject><subject>Adenoviruses</subject><subject>Animals</subject><subject>Antigens, CD</subject><subject>Antigens, Differentiation - genetics</subject><subject>Antigens, Differentiation - metabolism</subject><subject>beta-Galactosidase - biosynthesis</subject><subject>Bone marrow</subject><subject>Cells, Cultured</subject><subject>Cloning</subject><subject>CTLA-4 Antigen</subject><subject>Disease</subject><subject>DNA Primers - chemistry</subject><subject>DNA, Viral - metabolism</subject><subject>Enzymes</subject><subject>Gene Expression</subject><subject>Gene therapy</subject><subject>Genetic Therapy</subject><subject>Genetic Vectors</subject><subject>Genotype</subject><subject>Glucuronidase - genetics</subject><subject>Glucuronidase - 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Academic</collection><jtitle>Molecular therapy</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Kosuga, M</au><au>Takahashi, S</au><au>Sasaki, K</au><au>Li, X K</au><au>Fujino, M</au><au>Hamada, H</au><au>Suzuki, S</au><au>Yamada, M</au><au>Matsuo, N</au><au>Okuyama, T</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression</atitle><jtitle>Molecular therapy</jtitle><addtitle>Mol Ther</addtitle><date>2000-05</date><risdate>2000</risdate><volume>1</volume><issue>5 Pt 1</issue><spage>406</spage><epage>413</epage><pages>406-413</pages><issn>1525-0016</issn><eissn>1525-0024</eissn><abstract>Recombinant adenoviruses expressing human beta-glucuronidase (AxCAhGUS) and CTLA-4Ig (AxCACTLA-4Ig) were generated and therapeutic efficacy was investigated using a murine model of mucopolysaccharidosis type VII (MPSVII). Seven days after the intravenous administration of AxCAhGUS, high levels of beta-glucuronidase (GUSB) activity were observed in the liver, spleen, heart, lung, kidney, and serum, while viral DNA was predominantly detected in the liver. To investigate the contribution of in vivo cross-correction of GUSB between the liver and other organs, we injected the serum obtained from the transduced mice into untreated MPSVII mice. Similar distributions of GUSB activity were observed in the serum-injected mice, suggesting that GUSB activities detected in the extrahepatic organs were due to the cross-correction rather than the direct gene transduction. This result also suggested that maintaining high levels of GUSB in the systemic circulation was essential for the effective treatment of MPSVII. To achieve this, we injected AxCAhGUS and AxCACTLA-4Ig into MPSVII mice. Serum GUSB activity was sustained at high levels for more than 200 days and morphological normalization of the liver and spleen was observed for a year. This suggests that long-term therapeutic efficacy in visceral organs of MPSVII is achievable by coexpression of CTLA-4Ig through an in vivo cross-correction pathway.</abstract><cop>United States</cop><pub>Elsevier Limited</pub><pmid>10933961</pmid><doi>10.1006/mthe.2000.0067</doi><tpages>8</tpages><oa>free_for_read</oa></addata></record> |
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| ispartof | Molecular therapy, 2000-05, Vol.1 (5 Pt 1), p.406-413 |
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| subjects | Abatacept Adenoviridae - genetics Adenoviridae - metabolism Adenoviruses Animals Antigens, CD Antigens, Differentiation - genetics Antigens, Differentiation - metabolism beta-Galactosidase - biosynthesis Bone marrow Cells, Cultured Cloning CTLA-4 Antigen Disease DNA Primers - chemistry DNA, Viral - metabolism Enzymes Gene Expression Gene therapy Genetic Therapy Genetic Vectors Genotype Glucuronidase - genetics Glucuronidase - metabolism Humans Immunoconjugates Immunoglobulin Fc Fragments - metabolism Kidney - enzymology Kidney - pathology Liver Liver - enzymology Liver - pathology Lung - enzymology Lung - pathology Medical research Mice Mice, Inbred C57BL Mucopolysaccharidosis VII - genetics Mucopolysaccharidosis VII - metabolism Mucopolysaccharidosis VII - therapy Peptides Receptor, IGF Type 2 - metabolism Recombinant Fusion Proteins - genetics Recombinant Fusion Proteins - metabolism Research centers Spleen Spleen - enzymology Spleen - pathology |
| title | Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression |
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