tailieunhanh - Báo cáo sinh học: "Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice"

Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice | Genetic Vaccines and Therapy BioMed Central Short paper Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice Christina A Pacakh Yoshihisa Sakaih Bijoy D Thattaliyath Cathryn S Mah and Barry J Byrne Open Access Address Powell Gene Therapy Center College of Medicine University of Florida 1600 SW Archer Road Gainesville FL 32610-0266 USA Email Christina A Pacak - Yoshihisa Sakai - ysakai@ Bijoy D Thattaliyath - bijoy@ Cathryn S Mah - cmah@ Barry J Byrne - bbyrne@ Corresponding authors fEqual contributors Published 23 September 2008 Received 10 June 2008 . IAccepted 23 September 2008 Genetic Vaccines and Therapy 2008 6 13 doi l479-0556-6-l3 This article is available from http content 6 l l3 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 AAV9 capsid displays a high natural affinity for the heart following a single intravenous IV administration in both newborn and adult mice. It also results in substantial albeit relatively lower expression levels in many other tissues. To increase the overall safety of this gene delivery method we sought to identify which one of a group of promoters is able to confer the highest level of cardiac specific expression and concurrently which is able to provide a broad biodistribution of expression across both cardiac and skeletal muscle. The in vivo behavior of five different promoters was compared CMV desmin Des alpha-myosin heavy chain a-MHC myosin light chain 2 MLC-2 and cardiac troponin C cTnC . Following IV .

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