tailieunhanh - Báo cáo sinh học: "Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle"

Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle | Genetic Vaccines and Therapy BioMed Central Short paper Relative persistence of AAV serotype I vector genomes in dystrophic muscle Christina A Pacak1 2 Thomas Conlon1 2 Cathryn S Mah 1 3 and Barry J Byrne 1 2 3 Open Access Address 1Powell Gene Therapy Center University of Florida Gainesville FL USA 2Department of Pediatrics University of Florida Gainesville FL USA and 3Division of Cellular and Molecular Therapy Department of Pediatrics University of Florida Gainesville FL USA Email Christina A Pacak - Thomas Conlon - conlon@ Cathryn S Mah - cmah@ Barry J Byrne - bbyrne@ Corresponding authors Published 15 October 2008 Received 10 June 2008 . JI nrr X. Accepted 15 October 2008 Genetic Vaccines and Therapy 2008 6 14 doi l479-0556-6-l4 This article is available from http content 6 l l4 2008 Pacak et al licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License http licenses by which permits unrestricted use distribution and reproduction in any medium provided the original work is properly cited. Abstract The purpose of this study was to assess the behavior of pseudotyped recombinant adeno-associated virus type l rAAV2 l vector genomes in dystrophic skeletal muscle. A comparison was made between a therapeutic vector and a reporter vector by injecting the hindlimb in a mouse model of Limb Girdle Muscular Dystrophy Type 2D LGMD-2D prior to disease onset. We hypothesized that the therapeutic vector would establish long-term persistence through prevention of myofiber turnover. In contrast the reporter vector genome copy number would diminish over time due to disease-associated muscle degradation. One day old alpha sarcoglycan knockout mice sgca-1- were injected with l X l0ii vector genomes of rAAV2 l-tMCK-sgca in one hindlimb and the same dose of rAAV2 l-tMCK-LacZ in the contra lateral .