Gene therapy for murine glycerol kinase deficiency: Importance of murine ortholog
A glycerol kinase (Gyk) knock-out (KO) mouse model permits improved understanding of glycerol kinase (GK) deficiency (GKD) pathogenesis, however, early death of affected mice limits its utility. The purpose of this work was to delay death of affected males to investigate thoroughly their phenotypes....
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| Veröffentlicht in: | Biochemical and biophysical research communications 2005-09, Vol.335 (1), p.247-255 |
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| container_title | Biochemical and biophysical research communications |
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| creator | Kuwada, N. Nagano, K. MacLennan, N. Havens, J. Kumar, M. Dipple, K.M. McCabe, E.R.B. |
| description | A glycerol kinase (Gyk) knock-out (KO) mouse model permits improved understanding of glycerol kinase (GK) deficiency (GKD) pathogenesis, however, early death of affected mice limits its utility. The purpose of this work was to delay death of affected males to investigate thoroughly their phenotypes. An adenoviral vector carrying the human (Adeno-XGK) or mouse (Adeno-XGyk) GK gene was injected into KO mice within 24
h of birth. Adeno-XGK did not change KO mouse survival time despite liver GK activity greater than 100% of wild type. However, Adeno-XGyk improved KO mouse survival time greater than two-fold. These investigations demonstrate that gene replacement therapy for Gyk KO mice is more efficacious using murine Gyk than human GK. These studies expand our understanding of GKD pathogenesis in the murine model, and show that while murine GKD is more severe than in humans, GKD mice have similar metabolic disturbances to affected humans with hypoglycemia and acidemia. |
| doi_str_mv | 10.1016/j.bbrc.2005.07.066 |
| format | Article |
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h of birth. Adeno-XGK did not change KO mouse survival time despite liver GK activity greater than 100% of wild type. However, Adeno-XGyk improved KO mouse survival time greater than two-fold. These investigations demonstrate that gene replacement therapy for Gyk KO mice is more efficacious using murine Gyk than human GK. These studies expand our understanding of GKD pathogenesis in the murine model, and show that while murine GKD is more severe than in humans, GKD mice have similar metabolic disturbances to affected humans with hypoglycemia and acidemia.</description><identifier>ISSN: 0006-291X</identifier><identifier>EISSN: 1090-2104</identifier><identifier>DOI: 10.1016/j.bbrc.2005.07.066</identifier><identifier>PMID: 16105550</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Acidemia ; Adenoviridae - genetics ; Adenovirus ; Animals ; Blood Gas Analysis ; Blood Glucose - metabolism ; Body Weight ; Fatty Acids, Nonesterified - blood ; Gene therapy ; Genetic Therapy ; Glycerol - blood ; Glycerol kinase ; Glycerol Kinase - deficiency ; Glycerol Kinase - genetics ; Glycerol Kinase - metabolism ; Glycerol metabolism ; Hypoglycemia ; Mice ; Mice, Knockout ; Murine models ; Survival Rate</subject><ispartof>Biochemical and biophysical research communications, 2005-09, Vol.335 (1), p.247-255</ispartof><rights>2005 Elsevier Inc.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c385t-22e1687c72586a11e88d650e983dfa79701f404d901b991ae0a51679f0b93c13</citedby><cites>FETCH-LOGICAL-c385t-22e1687c72586a11e88d650e983dfa79701f404d901b991ae0a51679f0b93c13</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.bbrc.2005.07.066$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/16105550$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Kuwada, N.</creatorcontrib><creatorcontrib>Nagano, K.</creatorcontrib><creatorcontrib>MacLennan, N.</creatorcontrib><creatorcontrib>Havens, J.</creatorcontrib><creatorcontrib>Kumar, M.</creatorcontrib><creatorcontrib>Dipple, K.M.</creatorcontrib><creatorcontrib>McCabe, E.R.B.</creatorcontrib><title>Gene therapy for murine glycerol kinase deficiency: Importance of murine ortholog</title><title>Biochemical and biophysical research communications</title><addtitle>Biochem Biophys Res Commun</addtitle><description>A glycerol kinase (Gyk) knock-out (KO) mouse model permits improved understanding of glycerol kinase (GK) deficiency (GKD) pathogenesis, however, early death of affected mice limits its utility. The purpose of this work was to delay death of affected males to investigate thoroughly their phenotypes. An adenoviral vector carrying the human (Adeno-XGK) or mouse (Adeno-XGyk) GK gene was injected into KO mice within 24
h of birth. Adeno-XGK did not change KO mouse survival time despite liver GK activity greater than 100% of wild type. However, Adeno-XGyk improved KO mouse survival time greater than two-fold. These investigations demonstrate that gene replacement therapy for Gyk KO mice is more efficacious using murine Gyk than human GK. These studies expand our understanding of GKD pathogenesis in the murine model, and show that while murine GKD is more severe than in humans, GKD mice have similar metabolic disturbances to affected humans with hypoglycemia and acidemia.</description><subject>Acidemia</subject><subject>Adenoviridae - genetics</subject><subject>Adenovirus</subject><subject>Animals</subject><subject>Blood Gas Analysis</subject><subject>Blood Glucose - metabolism</subject><subject>Body Weight</subject><subject>Fatty Acids, Nonesterified - blood</subject><subject>Gene therapy</subject><subject>Genetic Therapy</subject><subject>Glycerol - blood</subject><subject>Glycerol kinase</subject><subject>Glycerol Kinase - deficiency</subject><subject>Glycerol Kinase - genetics</subject><subject>Glycerol Kinase - metabolism</subject><subject>Glycerol metabolism</subject><subject>Hypoglycemia</subject><subject>Mice</subject><subject>Mice, Knockout</subject><subject>Murine models</subject><subject>Survival Rate</subject><issn>0006-291X</issn><issn>1090-2104</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2005</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkEtLxDAUhYMoOj7-gAvpyl3rvX2kibgR8QWCCC7chTS9ncnYNmPSEebf23FG3OnqwOE7Z_ExdoqQICC_mCdV5U2SAhQJlAlwvsMmCBLiFCHfZRMA4HEq8e2AHYYwB0DMudxnB8gRiqKACXu5p56iYUZeL1ZR43zULb0dq2m7MuRdG73bXgeKamqssdSb1WX02C2cH3RvKHLNz2BsZq5102O21-g20Mk2j9jr3e3rzUP89Hz_eHP9FJtMFEOcpoRclKZMC8E1IglR8wJIiqxudClLwCaHvJaAlZSoCXSBvJQNVDIzmB2x883twruPJYVBdTYYalvdk1sGxUUuRIb5vyCWmRxlrMF0AxrvQvDUqIW3nfYrhaDWwtVcrYWrtXAFpRqFj6Oz7fuy6qj-nWwNj8DVBqDRxaclr8K3RqqtJzOo2tm__r8AkaSQ2Q</recordid><startdate>20050916</startdate><enddate>20050916</enddate><creator>Kuwada, N.</creator><creator>Nagano, K.</creator><creator>MacLennan, N.</creator><creator>Havens, J.</creator><creator>Kumar, M.</creator><creator>Dipple, K.M.</creator><creator>McCabe, E.R.B.</creator><general>Elsevier Inc</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>20050916</creationdate><title>Gene therapy for murine glycerol kinase deficiency: Importance of murine ortholog</title><author>Kuwada, N. ; Nagano, K. ; MacLennan, N. ; Havens, J. ; Kumar, M. ; Dipple, K.M. ; McCabe, E.R.B.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c385t-22e1687c72586a11e88d650e983dfa79701f404d901b991ae0a51679f0b93c13</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2005</creationdate><topic>Acidemia</topic><topic>Adenoviridae - genetics</topic><topic>Adenovirus</topic><topic>Animals</topic><topic>Blood Gas Analysis</topic><topic>Blood Glucose - metabolism</topic><topic>Body Weight</topic><topic>Fatty Acids, Nonesterified - blood</topic><topic>Gene therapy</topic><topic>Genetic Therapy</topic><topic>Glycerol - blood</topic><topic>Glycerol kinase</topic><topic>Glycerol Kinase - deficiency</topic><topic>Glycerol Kinase - genetics</topic><topic>Glycerol Kinase - metabolism</topic><topic>Glycerol metabolism</topic><topic>Hypoglycemia</topic><topic>Mice</topic><topic>Mice, Knockout</topic><topic>Murine models</topic><topic>Survival Rate</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Kuwada, N.</creatorcontrib><creatorcontrib>Nagano, K.</creatorcontrib><creatorcontrib>MacLennan, N.</creatorcontrib><creatorcontrib>Havens, J.</creatorcontrib><creatorcontrib>Kumar, M.</creatorcontrib><creatorcontrib>Dipple, K.M.</creatorcontrib><creatorcontrib>McCabe, E.R.B.</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>Biochemical and biophysical research communications</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Kuwada, N.</au><au>Nagano, K.</au><au>MacLennan, N.</au><au>Havens, J.</au><au>Kumar, M.</au><au>Dipple, K.M.</au><au>McCabe, E.R.B.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Gene therapy for murine glycerol kinase deficiency: Importance of murine ortholog</atitle><jtitle>Biochemical and biophysical research communications</jtitle><addtitle>Biochem Biophys Res Commun</addtitle><date>2005-09-16</date><risdate>2005</risdate><volume>335</volume><issue>1</issue><spage>247</spage><epage>255</epage><pages>247-255</pages><issn>0006-291X</issn><eissn>1090-2104</eissn><abstract>A glycerol kinase (Gyk) knock-out (KO) mouse model permits improved understanding of glycerol kinase (GK) deficiency (GKD) pathogenesis, however, early death of affected mice limits its utility. The purpose of this work was to delay death of affected males to investigate thoroughly their phenotypes. An adenoviral vector carrying the human (Adeno-XGK) or mouse (Adeno-XGyk) GK gene was injected into KO mice within 24
h of birth. Adeno-XGK did not change KO mouse survival time despite liver GK activity greater than 100% of wild type. However, Adeno-XGyk improved KO mouse survival time greater than two-fold. These investigations demonstrate that gene replacement therapy for Gyk KO mice is more efficacious using murine Gyk than human GK. These studies expand our understanding of GKD pathogenesis in the murine model, and show that while murine GKD is more severe than in humans, GKD mice have similar metabolic disturbances to affected humans with hypoglycemia and acidemia.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>16105550</pmid><doi>10.1016/j.bbrc.2005.07.066</doi><tpages>9</tpages></addata></record> |
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| subjects | Acidemia Adenoviridae - genetics Adenovirus Animals Blood Gas Analysis Blood Glucose - metabolism Body Weight Fatty Acids, Nonesterified - blood Gene therapy Genetic Therapy Glycerol - blood Glycerol kinase Glycerol Kinase - deficiency Glycerol Kinase - genetics Glycerol Kinase - metabolism Glycerol metabolism Hypoglycemia Mice Mice, Knockout Murine models Survival Rate |
| title | Gene therapy for murine glycerol kinase deficiency: Importance of murine ortholog |
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