TAILIEUCHUNG - Báo cáo sinh học: "Long term physiologic modification using rAAV in utero gene-therapy"

Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Long term physiologic modification using rAAV in utero gene-therapy | Genetic Vaccines and Therapy BioMed Central Research Long term physiologic modification using rAAV in utero gene-therapy Deiadra J Garrett1 2 J Craig Cohen 2 and Janet E Larson1 Open Access Address 1Ochsner Children s Research Institute Ochsner Clinic Foundation New Orleans LA 70121 USA and 2Departments of Medicine Biochemistry and Genetics Louisiana State University Health Sciences Center New Orleans LA 70112 USA Email Deiadra J Garrett - Djgarre@ J Craig Cohen - ccohen@ Janet E Larson - jlarson@ Corresponding author Published 19 May 2004 Received 04 February 2004 Accepted 19 May 2004 Genetic vaccines and Therapy 2004 2 4 This article is available from http content 2 l 4 2004 Garrett et al licensee BioMed Central Ltd. This is an Open Access article verbatim copying and redistribution of this article are permitted in all media for any purpose provided this notice is preserved along with the article s original URL. Abstract Background Transfer of genes in utero via the amniotic fluid was shown previously with recombinant adeno-associated viruses rAAV to be highly efficient. Expression for over one year was demonstrated using reporter genes. In addition it was shown previously that transgenes delivered by this method release protein into the general circulation. Given these results experiments were designed to test the hypothesis that in utero rAAV gene therapy could result in long term physiologic modification. Methods A rAAV recombinant expressing ciliary neurotrophic factor cntf and green fluorescent gfp in a polycistronic messenger was used to treat rat fetuses in utero. CNTF causes weight loss and decreased water consumption as a measurable physiologic effect. GFP was used as a marker of gene expression. Results In utero gene transfer with rAAV carrying human cntf and gfp resulted in long-term gene expression in rat. CNTF-specific physiologic effects of a decrease in weight and water intake were obtained. .

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