TAILIEUCHUNG - Báo cáo sinh học: " Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats"

Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats | Genetic Vaccines and Therapy BioMed Central Research Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats Tracey Graham Jenny McIntosh2 Lorraine M Work1 Amit Nathwani2 and Andrew H Baker 1 Open Access Address 1British Heart Foundation Glasgow Cardiovascular Research Centre University of Glasgow Glasgow G12 8TA UK and 2Department of Haematology University College London London UK Email Tracey Graham - tlg2g@ Jenny McIntosh - Lorraine M Work - lmw3u@ AmitNathwani - Andrew H Baker - ab11f@ Corresponding author Published 3 March 2008 Received 28 January 2008 Genetic Vaccines and Therapy 2008 6 9 doi 186 1479-0556-6-9 Accepted 3 March 2008 This article is available from http content 6 1 9 2008 Graham 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 Background Vectors based on adeno-associated virus-8 AAV8 have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection particularly in relation to haemophilia gene therapy. AAV8 has also been proposed for gene therapy targeted at skeletal and cardiac muscle again via intravascular injection. It is important to assess vector targeting at the level of virion accumulation and transgene expression in multiple species to ascertain potential issues relating to species variation in infectivity profiles. Methods We used AAV8 vectors expressing human factor IX FIX from the liver-specific LP-I promoter and administered this virus via the intravascular route of injection into 12 week old Wistar Kyoto rats. We assessed FIX levels in serum by

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