期刊
HUMAN GENE THERAPY
卷 20, 期 7, 页码 759-766出版社
MARY ANN LIEBERT, INC
DOI: 10.1089/hum.2008.197
关键词
-
资金
- NIH [2R01 AR18860, 1P01 NS046788]
- Muscular Dystrophy Association
- [1R21 DK073175]
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 (p < 0.05) increase in body length and body weight, without reaching full normalization, and significant (p < 0.05) 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.
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