Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector
Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encodi...
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| Veröffentlicht in: | Molecular therapy 2011-11, Vol.19 (11), p.1961-1970 |
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| creator | Luo, Xiaoyan Hall, Gentzon Li, Songtao Bird, Andrew Lavin, Peter J Winn, Michelle P Kemper, Alex R Brown, Talmage T Koeberl, Dwight D |
| description | Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encoding human G6Pase was pseudotyped with four serotypes, AAV2, AAV7, AAV8, and AAV9, and we evaluated efficacy in 12-day-old G6pase (−/−) mice. Hypoglycemia during fasting (plasma glucose 6 months by the dsAAV2/7, dsAAV2/8, and dsAAV2/9 vectors. Prolonged fasting for 8 hours revealed normalization of blood glucose following dsAAV2/9 vector administration at the higher dose. The glycogen content of kidney was reduced by >65% with both the dsAAV2/7 and dsAAV2/9 vectors, and renal glycogen content was stably reduced between 7 and 12 months of age for the dsAAV2/9 vector-treated mice. Every vector-treated group had significantly reduced glycogen content in the liver, in comparison with untreated G6pase (−/−) mice. G6Pase was expressed in many renal epithelial cells of with the dsAAV2/9 vector for up to 12 months. Albuminuria and renal fibrosis were reduced by the dsAAV2/9 vector. Hepatorenal correction in G6pase (−/−) mice demonstrates the potential of AAV vectors for the correction of inherited diseases of metabolism. |
| doi_str_mv | 10.1038/mt.2011.126 |
| format | Article |
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Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encoding human G6Pase was pseudotyped with four serotypes, AAV2, AAV7, AAV8, and AAV9, and we evaluated efficacy in 12-day-old G6pase (−/−) mice. Hypoglycemia during fasting (plasma glucose <100 mg/dl) was prevented for >6 months by the dsAAV2/7, dsAAV2/8, and dsAAV2/9 vectors. Prolonged fasting for 8 hours revealed normalization of blood glucose following dsAAV2/9 vector administration at the higher dose. The glycogen content of kidney was reduced by >65% with both the dsAAV2/7 and dsAAV2/9 vectors, and renal glycogen content was stably reduced between 7 and 12 months of age for the dsAAV2/9 vector-treated mice. Every vector-treated group had significantly reduced glycogen content in the liver, in comparison with untreated G6pase (−/−) mice. G6Pase was expressed in many renal epithelial cells of with the dsAAV2/9 vector for up to 12 months. Albuminuria and renal fibrosis were reduced by the dsAAV2/9 vector. Hepatorenal correction in G6pase (−/−) mice demonstrates the potential of AAV vectors for the correction of inherited diseases of metabolism.</description><identifier>ISSN: 1525-0016</identifier><identifier>EISSN: 1525-0024</identifier><identifier>DOI: 10.1038/mt.2011.126</identifier><identifier>PMID: 21730973</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adeno-associated virus ; Age ; Animals ; Cholesterol ; Dependovirus - genetics ; Disease Models, Animal ; Drug dosages ; Female ; Gene Expression Regulation ; Gene therapy ; Genetic Therapy ; Genetic Vectors - administration & dosage ; Genetic Vectors - genetics ; Genetics ; Glucose ; Glucose-6-Phosphatase - genetics ; Glycogen Storage Disease Type I - genetics ; Glycogen Storage Disease Type I - mortality ; Glycogen Storage Disease Type I - therapy ; Humans ; Hypoglycemia ; Hypoglycemia - genetics ; Hypoglycemia - therapy ; Kaplan-Meier Estimate ; Kidney - metabolism ; Kidney diseases ; Liver - metabolism ; Liver cancer ; Male ; Metabolism ; Mice ; Mice, Knockout ; Original ; Pediatrics ; Phosphatase ; Plasma ; Survival analysis ; Tumors ; Vectors (Biology)</subject><ispartof>Molecular therapy, 2011-11, Vol.19 (11), p.1961-1970</ispartof><rights>2011 The American Society of Gene & Cell Therapy</rights><rights>Copyright Nature Publishing Group Nov 2011</rights><rights>Copyright © 2011 The American Society of Gene & Cell Therapy 2011 The American Society of Gene & Cell Therapy</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c487t-86b219cec6e961e74eb1f6d09dcefe1d5639ba13ae55c4560f45df182f7ad0e13</citedby><cites>FETCH-LOGICAL-c487t-86b219cec6e961e74eb1f6d09dcefe1d5639ba13ae55c4560f45df182f7ad0e13</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222521/pdf/$$EPDF$$P50$$Gpubmedcentral$$H</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222521/$$EHTML$$P50$$Gpubmedcentral$$H</linktohtml><link.rule.ids>230,314,723,776,780,881,27901,27902,53766,53768</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/21730973$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Luo, Xiaoyan</creatorcontrib><creatorcontrib>Hall, Gentzon</creatorcontrib><creatorcontrib>Li, Songtao</creatorcontrib><creatorcontrib>Bird, Andrew</creatorcontrib><creatorcontrib>Lavin, Peter J</creatorcontrib><creatorcontrib>Winn, Michelle P</creatorcontrib><creatorcontrib>Kemper, Alex R</creatorcontrib><creatorcontrib>Brown, Talmage T</creatorcontrib><creatorcontrib>Koeberl, Dwight D</creatorcontrib><title>Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector</title><title>Molecular therapy</title><addtitle>Mol Ther</addtitle><description>Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encoding human G6Pase was pseudotyped with four serotypes, AAV2, AAV7, AAV8, and AAV9, and we evaluated efficacy in 12-day-old G6pase (−/−) mice. Hypoglycemia during fasting (plasma glucose <100 mg/dl) was prevented for >6 months by the dsAAV2/7, dsAAV2/8, and dsAAV2/9 vectors. Prolonged fasting for 8 hours revealed normalization of blood glucose following dsAAV2/9 vector administration at the higher dose. The glycogen content of kidney was reduced by >65% with both the dsAAV2/7 and dsAAV2/9 vectors, and renal glycogen content was stably reduced between 7 and 12 months of age for the dsAAV2/9 vector-treated mice. Every vector-treated group had significantly reduced glycogen content in the liver, in comparison with untreated G6pase (−/−) mice. G6Pase was expressed in many renal epithelial cells of with the dsAAV2/9 vector for up to 12 months. Albuminuria and renal fibrosis were reduced by the dsAAV2/9 vector. Hepatorenal correction in G6pase (−/−) mice demonstrates the potential of AAV vectors for the correction of inherited diseases of metabolism.</description><subject>Adeno-associated virus</subject><subject>Age</subject><subject>Animals</subject><subject>Cholesterol</subject><subject>Dependovirus - genetics</subject><subject>Disease Models, Animal</subject><subject>Drug dosages</subject><subject>Female</subject><subject>Gene Expression Regulation</subject><subject>Gene therapy</subject><subject>Genetic Therapy</subject><subject>Genetic Vectors - administration & dosage</subject><subject>Genetic Vectors - genetics</subject><subject>Genetics</subject><subject>Glucose</subject><subject>Glucose-6-Phosphatase - genetics</subject><subject>Glycogen Storage Disease Type I - genetics</subject><subject>Glycogen Storage Disease Type I - mortality</subject><subject>Glycogen Storage Disease Type I - therapy</subject><subject>Humans</subject><subject>Hypoglycemia</subject><subject>Hypoglycemia - genetics</subject><subject>Hypoglycemia - therapy</subject><subject>Kaplan-Meier Estimate</subject><subject>Kidney - metabolism</subject><subject>Kidney diseases</subject><subject>Liver - metabolism</subject><subject>Liver cancer</subject><subject>Male</subject><subject>Metabolism</subject><subject>Mice</subject><subject>Mice, Knockout</subject><subject>Original</subject><subject>Pediatrics</subject><subject>Phosphatase</subject><subject>Plasma</subject><subject>Survival analysis</subject><subject>Tumors</subject><subject>Vectors (Biology)</subject><issn>1525-0016</issn><issn>1525-0024</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2011</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><recordid>eNp9kcFrFDEUhwdRbK2evEvAg4LMmpdMMjMXoWxrW6h4sNZjyCRvtimzyTaZKex_34xbFxXxlEfelx_v5SuK10AXQHnzcT0uGAVYAJNPikMQTJSUsurpvgZ5ULxI6TZXIFr5vDhgUHPa1vywmM5xo8cQ0euBLEOMaEYXPHGefJmi80jOhq0JK_TkW8b0CsmJS6gTkqvtBskF-eHGG6LJSZi6Acs0Ru0tWnJs0YdSpxSM02O-uHZxSuQ654f4snjW6yHhq8fzqPj--fRqeV5efj27WB5flqZq6rFsZMegNWgkthKwrrCDXlraWoM9ghWSt50GrlEIUwlJ-0rYHhrW19pSBH5UfNrlbqZujfmVz-MNahPdWsetCtqpPzve3ahVuFecMSbYHPDuMSCGuwnTqNYuGRwG7TFMSbWUcc7pT_L9f0mQsmGyoYJm9O1f6G2YYhaQqbqFtgHaztSHHWViSClivx8bqJrFq_WoZvEqi8_0m9833bO_TGdA7ADM_33vMKpkHHqD1s3OlQ3un8EPnDC8Lw</recordid><startdate>20111101</startdate><enddate>20111101</enddate><creator>Luo, Xiaoyan</creator><creator>Hall, Gentzon</creator><creator>Li, Songtao</creator><creator>Bird, Andrew</creator><creator>Lavin, Peter J</creator><creator>Winn, Michelle P</creator><creator>Kemper, Alex R</creator><creator>Brown, Talmage T</creator><creator>Koeberl, Dwight D</creator><general>Elsevier Inc</general><general>Elsevier Limited</general><general>Nature Publishing Group</general><scope>6I.</scope><scope>AAFTH</scope><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>3V.</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M7P</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>7QO</scope><scope>8FD</scope><scope>FR3</scope><scope>P64</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20111101</creationdate><title>Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector</title><author>Luo, Xiaoyan ; Hall, Gentzon ; Li, Songtao ; Bird, Andrew ; Lavin, Peter J ; Winn, Michelle P ; Kemper, Alex R ; Brown, Talmage T ; Koeberl, Dwight D</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c487t-86b219cec6e961e74eb1f6d09dcefe1d5639ba13ae55c4560f45df182f7ad0e13</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2011</creationdate><topic>Adeno-associated virus</topic><topic>Age</topic><topic>Animals</topic><topic>Cholesterol</topic><topic>Dependovirus - 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Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Molecular therapy</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Luo, Xiaoyan</au><au>Hall, Gentzon</au><au>Li, Songtao</au><au>Bird, Andrew</au><au>Lavin, Peter J</au><au>Winn, Michelle P</au><au>Kemper, Alex R</au><au>Brown, Talmage T</au><au>Koeberl, Dwight D</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector</atitle><jtitle>Molecular therapy</jtitle><addtitle>Mol Ther</addtitle><date>2011-11-01</date><risdate>2011</risdate><volume>19</volume><issue>11</issue><spage>1961</spage><epage>1970</epage><pages>1961-1970</pages><issn>1525-0016</issn><eissn>1525-0024</eissn><abstract>Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encoding human G6Pase was pseudotyped with four serotypes, AAV2, AAV7, AAV8, and AAV9, and we evaluated efficacy in 12-day-old G6pase (−/−) mice. Hypoglycemia during fasting (plasma glucose <100 mg/dl) was prevented for >6 months by the dsAAV2/7, dsAAV2/8, and dsAAV2/9 vectors. Prolonged fasting for 8 hours revealed normalization of blood glucose following dsAAV2/9 vector administration at the higher dose. The glycogen content of kidney was reduced by >65% with both the dsAAV2/7 and dsAAV2/9 vectors, and renal glycogen content was stably reduced between 7 and 12 months of age for the dsAAV2/9 vector-treated mice. Every vector-treated group had significantly reduced glycogen content in the liver, in comparison with untreated G6pase (−/−) mice. G6Pase was expressed in many renal epithelial cells of with the dsAAV2/9 vector for up to 12 months. Albuminuria and renal fibrosis were reduced by the dsAAV2/9 vector. Hepatorenal correction in G6pase (−/−) mice demonstrates the potential of AAV vectors for the correction of inherited diseases of metabolism.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>21730973</pmid><doi>10.1038/mt.2011.126</doi><tpages>10</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Adeno-associated virus Age Animals Cholesterol Dependovirus - genetics Disease Models, Animal Drug dosages Female Gene Expression Regulation Gene therapy Genetic Therapy Genetic Vectors - administration & dosage Genetic Vectors - genetics Genetics Glucose Glucose-6-Phosphatase - genetics Glycogen Storage Disease Type I - genetics Glycogen Storage Disease Type I - mortality Glycogen Storage Disease Type I - therapy Humans Hypoglycemia Hypoglycemia - genetics Hypoglycemia - therapy Kaplan-Meier Estimate Kidney - metabolism Kidney diseases Liver - metabolism Liver cancer Male Metabolism Mice Mice, Knockout Original Pediatrics Phosphatase Plasma Survival analysis Tumors Vectors (Biology) |
| title | Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector |
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