{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T15:02:07Z","timestamp":1790521327118,"version":"4.1.0"},"reference-count":31,"publisher":"SAGE Publications","issue":"7","license":[{"start":{"date-parts":[[2009,7,1]],"date-time":"2009-07-01T00:00:00Z","timestamp":1246406400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2009,7]]},"abstract":"<jats:p>\n                    Growth hormone (GH) deficiency (GHD) causes somatic growth impairment. GH has a short half-life and therefore it must be administered by daily subcutaneous injections. Adeno-associated viral (AAV) vectors have been used to deliver genes to animals, and double-stranded AAV (dsAAV) vectors provide widespread and stable transgene expression. In the present study we tested whether an intramuscular injection of dsAAV vector expressing GH under the control of a muscle creatine kinase regulatory cassette would ensure sufficient systemic GH delivery in conjunction with muscle-specific expression. Virus-injected GHD mice showed a significant (\n                    <jats:italic toggle=\"yes\">p<\/jats:italic>\n                    \u2009&lt;\u20090.05) increase in body length and body weight, without reaching full normalization, and significant (\n                    <jats:italic toggle=\"yes\">p<\/jats:italic>\n                    \u2009&lt;\u20090.05) reduction in absolute and relative visceral fat. Quantitative RT-PCR showed preferential GH expression in skeletal muscles that was confirmed by qualitative fluorescence analysis in mice injected with a similar virus expressing green fluorescent protein. The present study shows that systemic GH delivery to GHD animals is possible via a single intramuscular injection of dsAAV carrying a muscle-specific GH-expressing regulatory cassette.\n                  <\/jats:p>","DOI":"10.1089\/hum.2008.197","type":"journal-article","created":{"date-parts":[[2009,3,19]],"date-time":"2009-03-19T10:18:23Z","timestamp":1237457903000},"page":"759-766","source":"Crossref","is-referenced-by-count":27,"title":["Partial Rescue of Growth Failure in Growth Hormone (GH)-Deficient Mice by a Single Injection of a Double-Stranded Adeno-Associated Viral Vector Expressing the GH Gene Driven by a Muscle-Specific Regulatory Cassette"],"prefix":"10.1177","volume":"20","author":[{"given":"Marco","family":"Martari","sequence":"first","affiliation":[{"name":"Johns Hopkins University School of Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessia","family":"Sagazio","sequence":"additional","affiliation":[{"name":"Johns Hopkins University School of Medicine"},{"name":"University G. D'Annunzio","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ali","family":"Mohamadi","sequence":"additional","affiliation":[{"name":"Johns Hopkins University School of Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Quynh","family":"Nguyen","sequence":"additional","affiliation":[{"name":"University of Washington"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen D.","family":"Hauschka","sequence":"additional","affiliation":[{"name":"University of Washington"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eun","family":"Kim","sequence":"additional","affiliation":[{"name":"University of Pittsburgh"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Salvatori","sequence":"additional","affiliation":[{"name":"Johns Hopkins University School of Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,3,5]]},"reference":[{"key":"e_1_3_3_2_1","doi-asserted-by":"publisher","DOI":"10.1210\/en.2004-0119"},{"key":"e_1_3_3_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ghir.2005.05.001"},{"key":"e_1_3_3_4_1","first-page":"1","article-title":"Clinical effectiveness and cost-effectiveness of growth hormone in children: A systematic review and economic evaluation","volume":"6","author":"Bryant J.","year":"2002","unstructured":"Bryant J., , Cave C., , Mihaylova B., , Chase D., , McIntyre L., , Gerard K., , Milne R. 2002. Clinical effectiveness and cost-effectiveness of growth hormone in children: A systematic review and economic evaluation. Health Technol. Assess., 6:1\u2013168.","journal-title":"Health Technol. 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