Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector
Limb-girdle muscular dystrophies 2C–F represent a family of autosomal recessive diseases caused by defects in sarcoglycan genes 1 . The cardiomyopathic hamster is a naturally occurring model for limb-girdle muscular dystrophy caused by a primary deficiency in δ-sarcoglycan 2 , 3 , 4 , 5 . We show he...
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| Veröffentlicht in: | Nature medicine 1999-04, Vol.5 (4), p.439-443 |
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| creator | Greelish, James P. Su, Leonard T. Lankford, Edward B. Burkman, James M. Chen, Haiyan Konig, Stephane K. Mercier, Isabelle M. Desjardins, Philippe R. Mitchell, Marilyn A. Zheng, Xiang guang Leferovich, John Gao, Guang Ping Balice-Gordon, Rita J. Wilson, James M. Stedman, Hansell H. |
| description | Limb-girdle muscular dystrophies 2C–F represent a family of autosomal recessive diseases caused by defects in sarcoglycan genes
1
. The cardiomyopathic hamster is a naturally occurring model for limb-girdle muscular dystrophy caused by a primary deficiency in δ-sarcoglycan
2
,
3
,
4
,
5
. We show here that acute sarcolemmal disruption occurs in this animal model during forceful muscle contraction. A recombinant adeno-associated virus vector encoding human δ-sarcoglycan conferred efficient and stable genetic reconstitution in the adult cardiomyopathic hamster when injected directly into muscle. A quantitative assay demonstrated that vector-transduced muscle fibers are stably protected from sarcolemmal disruption; there was no associated inflammation or immunologic response to the vector-encoded protein. Efficient gene transduction with rescue of the sarcoglycan complex in muscle fibers of the distal hindlimb was also obtained after infusion of recombinant adeno-associated virus into the femoral artery in conjunction with histamine-induced endothelial permeabilization. This study provides a strong rationale for the development of gene therapy for limb-girdle muscular dystrophy. |
| doi_str_mv | 10.1038/7439 |
| format | Article |
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1
. The cardiomyopathic hamster is a naturally occurring model for limb-girdle muscular dystrophy caused by a primary deficiency in δ-sarcoglycan
2
,
3
,
4
,
5
. We show here that acute sarcolemmal disruption occurs in this animal model during forceful muscle contraction. A recombinant adeno-associated virus vector encoding human δ-sarcoglycan conferred efficient and stable genetic reconstitution in the adult cardiomyopathic hamster when injected directly into muscle. A quantitative assay demonstrated that vector-transduced muscle fibers are stably protected from sarcolemmal disruption; there was no associated inflammation or immunologic response to the vector-encoded protein. Efficient gene transduction with rescue of the sarcoglycan complex in muscle fibers of the distal hindlimb was also obtained after infusion of recombinant adeno-associated virus into the femoral artery in conjunction with histamine-induced endothelial permeabilization. This study provides a strong rationale for the development of gene therapy for limb-girdle muscular dystrophy.</description><identifier>ISSN: 1078-8956</identifier><identifier>EISSN: 1546-170X</identifier><identifier>DOI: 10.1038/7439</identifier><identifier>PMID: 10202936</identifier><language>eng</language><publisher>New York: Nature Publishing Group US</publisher><subject>Adeno-associated virus ; Animals ; Biomedical and Life Sciences ; Biomedicine ; Cancer Research ; Cell Membrane Permeability ; Cricetinae ; Cytoskeletal Proteins - genetics ; Cytoskeletal Proteins - therapeutic use ; Dependovirus - genetics ; Genetic Therapy - methods ; Genetic Vectors ; Histamine - therapeutic use ; Humans ; Infectious Diseases ; Membrane Glycoproteins - genetics ; Membrane Glycoproteins - therapeutic use ; Metabolic Diseases ; Molecular Medicine ; Muscular Dystrophy, Animal - therapy ; Neurosciences ; Perfusion ; Rats ; Rats, Inbred F344 ; Recombinant Proteins - therapeutic use ; Sarcoglycans ; Sarcolemma - pathology</subject><ispartof>Nature medicine, 1999-04, Vol.5 (4), p.439-443</ispartof><rights>Nature America Inc. 1999</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c332t-79bbf4f866d71d20442e252e900b976a054b704b974ab26a07af668e0c6b01b23</citedby><cites>FETCH-LOGICAL-c332t-79bbf4f866d71d20442e252e900b976a054b704b974ab26a07af668e0c6b01b23</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1038/7439$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1038/7439$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,776,780,27903,27904,41467,42536,51297</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/10202936$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Greelish, James P.</creatorcontrib><creatorcontrib>Su, Leonard T.</creatorcontrib><creatorcontrib>Lankford, Edward B.</creatorcontrib><creatorcontrib>Burkman, James M.</creatorcontrib><creatorcontrib>Chen, Haiyan</creatorcontrib><creatorcontrib>Konig, Stephane K.</creatorcontrib><creatorcontrib>Mercier, Isabelle M.</creatorcontrib><creatorcontrib>Desjardins, Philippe R.</creatorcontrib><creatorcontrib>Mitchell, Marilyn A.</creatorcontrib><creatorcontrib>Zheng, Xiang guang</creatorcontrib><creatorcontrib>Leferovich, John</creatorcontrib><creatorcontrib>Gao, Guang Ping</creatorcontrib><creatorcontrib>Balice-Gordon, Rita J.</creatorcontrib><creatorcontrib>Wilson, James M.</creatorcontrib><creatorcontrib>Stedman, Hansell H.</creatorcontrib><title>Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector</title><title>Nature medicine</title><addtitle>Nat Med</addtitle><addtitle>Nat Med</addtitle><description>Limb-girdle muscular dystrophies 2C–F represent a family of autosomal recessive diseases caused by defects in sarcoglycan genes
1
. The cardiomyopathic hamster is a naturally occurring model for limb-girdle muscular dystrophy caused by a primary deficiency in δ-sarcoglycan
2
,
3
,
4
,
5
. We show here that acute sarcolemmal disruption occurs in this animal model during forceful muscle contraction. A recombinant adeno-associated virus vector encoding human δ-sarcoglycan conferred efficient and stable genetic reconstitution in the adult cardiomyopathic hamster when injected directly into muscle. A quantitative assay demonstrated that vector-transduced muscle fibers are stably protected from sarcolemmal disruption; there was no associated inflammation or immunologic response to the vector-encoded protein. Efficient gene transduction with rescue of the sarcoglycan complex in muscle fibers of the distal hindlimb was also obtained after infusion of recombinant adeno-associated virus into the femoral artery in conjunction with histamine-induced endothelial permeabilization. This study provides a strong rationale for the development of gene therapy for limb-girdle muscular dystrophy.</description><subject>Adeno-associated virus</subject><subject>Animals</subject><subject>Biomedical and Life Sciences</subject><subject>Biomedicine</subject><subject>Cancer Research</subject><subject>Cell Membrane Permeability</subject><subject>Cricetinae</subject><subject>Cytoskeletal Proteins - genetics</subject><subject>Cytoskeletal Proteins - therapeutic use</subject><subject>Dependovirus - genetics</subject><subject>Genetic Therapy - methods</subject><subject>Genetic Vectors</subject><subject>Histamine - therapeutic use</subject><subject>Humans</subject><subject>Infectious Diseases</subject><subject>Membrane Glycoproteins - genetics</subject><subject>Membrane Glycoproteins - therapeutic use</subject><subject>Metabolic Diseases</subject><subject>Molecular Medicine</subject><subject>Muscular Dystrophy, Animal - therapy</subject><subject>Neurosciences</subject><subject>Perfusion</subject><subject>Rats</subject><subject>Rats, Inbred F344</subject><subject>Recombinant Proteins - therapeutic use</subject><subject>Sarcoglycans</subject><subject>Sarcolemma - pathology</subject><issn>1078-8956</issn><issn>1546-170X</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>1999</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkc1u1TAQhS1ERUvLKyAvgF3o2EmceImq8iNVYgFI7KKxM-l1ldjBdgr3FfrU9dXtAlZ447HmO2fkOYxdCHgvoO4vu6bWz9iZaBtViQ5-Pi81dH3V61adspcp3QFADa1-wU4FSJC6Vmfs4VtGMxOPlHKImF3wPEw874gnjDbcznuLntuwrDP94c7zcZ9yDOvOWb5syRbtSnHaEo38t8s7vnMp4-I8cfQjx-JcxMZ59JnjSD5UmFKwDnNR3LuIM78nW4ZfsJMJ50Svnu5z9uPj9ferz9XN109frj7cVLauZa46bczUTL1SYydGCU0jSbaSNIDRnUJoG9NBU-oGjSzvDielegKrDAgj63P27ui7xvBrK_8eFpcszTN6ClsalFa9Elr_FxSdLEccwDdH0MaQUqRpWKNbMO4HAcMhnOEQTsFeP_ltZqHxL-iYRgHeHoFUWv6W4nAXtujLMv41egSKx5eP</recordid><startdate>19990401</startdate><enddate>19990401</enddate><creator>Greelish, James P.</creator><creator>Su, Leonard T.</creator><creator>Lankford, Edward B.</creator><creator>Burkman, James M.</creator><creator>Chen, Haiyan</creator><creator>Konig, Stephane K.</creator><creator>Mercier, Isabelle M.</creator><creator>Desjardins, Philippe R.</creator><creator>Mitchell, Marilyn A.</creator><creator>Zheng, Xiang guang</creator><creator>Leferovich, John</creator><creator>Gao, Guang Ping</creator><creator>Balice-Gordon, Rita J.</creator><creator>Wilson, James M.</creator><creator>Stedman, Hansell H.</creator><general>Nature Publishing Group US</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7QO</scope><scope>8FD</scope><scope>FR3</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope></search><sort><creationdate>19990401</creationdate><title>Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector</title><author>Greelish, James P. ; Su, Leonard T. ; Lankford, Edward B. ; Burkman, James M. ; Chen, Haiyan ; Konig, Stephane K. ; Mercier, Isabelle M. ; Desjardins, Philippe R. ; Mitchell, Marilyn A. ; Zheng, Xiang guang ; Leferovich, John ; Gao, Guang Ping ; Balice-Gordon, Rita J. ; Wilson, James M. ; Stedman, Hansell H.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c332t-79bbf4f866d71d20442e252e900b976a054b704b974ab26a07af668e0c6b01b23</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>1999</creationdate><topic>Adeno-associated virus</topic><topic>Animals</topic><topic>Biomedical and Life Sciences</topic><topic>Biomedicine</topic><topic>Cancer Research</topic><topic>Cell Membrane Permeability</topic><topic>Cricetinae</topic><topic>Cytoskeletal Proteins - genetics</topic><topic>Cytoskeletal Proteins - therapeutic use</topic><topic>Dependovirus - genetics</topic><topic>Genetic Therapy - methods</topic><topic>Genetic Vectors</topic><topic>Histamine - therapeutic use</topic><topic>Humans</topic><topic>Infectious Diseases</topic><topic>Membrane Glycoproteins - genetics</topic><topic>Membrane Glycoproteins - therapeutic use</topic><topic>Metabolic Diseases</topic><topic>Molecular Medicine</topic><topic>Muscular Dystrophy, Animal - therapy</topic><topic>Neurosciences</topic><topic>Perfusion</topic><topic>Rats</topic><topic>Rats, Inbred F344</topic><topic>Recombinant Proteins - therapeutic use</topic><topic>Sarcoglycans</topic><topic>Sarcolemma - pathology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Greelish, James P.</creatorcontrib><creatorcontrib>Su, Leonard T.</creatorcontrib><creatorcontrib>Lankford, Edward B.</creatorcontrib><creatorcontrib>Burkman, James M.</creatorcontrib><creatorcontrib>Chen, Haiyan</creatorcontrib><creatorcontrib>Konig, Stephane K.</creatorcontrib><creatorcontrib>Mercier, Isabelle M.</creatorcontrib><creatorcontrib>Desjardins, Philippe R.</creatorcontrib><creatorcontrib>Mitchell, Marilyn A.</creatorcontrib><creatorcontrib>Zheng, Xiang guang</creatorcontrib><creatorcontrib>Leferovich, John</creatorcontrib><creatorcontrib>Gao, Guang Ping</creatorcontrib><creatorcontrib>Balice-Gordon, Rita J.</creatorcontrib><creatorcontrib>Wilson, James M.</creatorcontrib><creatorcontrib>Stedman, Hansell H.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Biotechnology Research Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Nature medicine</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Greelish, James P.</au><au>Su, Leonard T.</au><au>Lankford, Edward B.</au><au>Burkman, James M.</au><au>Chen, Haiyan</au><au>Konig, Stephane K.</au><au>Mercier, Isabelle M.</au><au>Desjardins, Philippe R.</au><au>Mitchell, Marilyn A.</au><au>Zheng, Xiang guang</au><au>Leferovich, John</au><au>Gao, Guang Ping</au><au>Balice-Gordon, Rita J.</au><au>Wilson, James M.</au><au>Stedman, Hansell H.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector</atitle><jtitle>Nature medicine</jtitle><stitle>Nat Med</stitle><addtitle>Nat Med</addtitle><date>1999-04-01</date><risdate>1999</risdate><volume>5</volume><issue>4</issue><spage>439</spage><epage>443</epage><pages>439-443</pages><issn>1078-8956</issn><eissn>1546-170X</eissn><abstract>Limb-girdle muscular dystrophies 2C–F represent a family of autosomal recessive diseases caused by defects in sarcoglycan genes
1
. The cardiomyopathic hamster is a naturally occurring model for limb-girdle muscular dystrophy caused by a primary deficiency in δ-sarcoglycan
2
,
3
,
4
,
5
. We show here that acute sarcolemmal disruption occurs in this animal model during forceful muscle contraction. A recombinant adeno-associated virus vector encoding human δ-sarcoglycan conferred efficient and stable genetic reconstitution in the adult cardiomyopathic hamster when injected directly into muscle. A quantitative assay demonstrated that vector-transduced muscle fibers are stably protected from sarcolemmal disruption; there was no associated inflammation or immunologic response to the vector-encoded protein. Efficient gene transduction with rescue of the sarcoglycan complex in muscle fibers of the distal hindlimb was also obtained after infusion of recombinant adeno-associated virus into the femoral artery in conjunction with histamine-induced endothelial permeabilization. This study provides a strong rationale for the development of gene therapy for limb-girdle muscular dystrophy.</abstract><cop>New York</cop><pub>Nature Publishing Group US</pub><pmid>10202936</pmid><doi>10.1038/7439</doi><tpages>5</tpages></addata></record> |
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| ispartof | Nature medicine, 1999-04, Vol.5 (4), p.439-443 |
| issn | 1078-8956 1546-170X |
| language | eng |
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| source | MEDLINE; Springer Nature - Complete Springer Journals; Springer Nature - Connect here FIRST to enable access |
| subjects | Adeno-associated virus Animals Biomedical and Life Sciences Biomedicine Cancer Research Cell Membrane Permeability Cricetinae Cytoskeletal Proteins - genetics Cytoskeletal Proteins - therapeutic use Dependovirus - genetics Genetic Therapy - methods Genetic Vectors Histamine - therapeutic use Humans Infectious Diseases Membrane Glycoproteins - genetics Membrane Glycoproteins - therapeutic use Metabolic Diseases Molecular Medicine Muscular Dystrophy, Animal - therapy Neurosciences Perfusion Rats Rats, Inbred F344 Recombinant Proteins - therapeutic use Sarcoglycans Sarcolemma - pathology |
| title | Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector |
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