tailieunhanh - Báo cáo sinh học: "Induction of revertant fibres in the mdx mouse using antisense oligonucleotides"

Tuyển tập các báo cáo nghiên cứu về sinh học được đăng trên tạp chí sinh học Journal of Biology đề tài: Induction of revertant fibres in the mdx mouse using antisense oligonucleotides | Genetic Vaccines and Therapy BioMed Central Research Open Access Induction of revertant fibres in the mdx mouse using antisense oligonucleotides Abbie M Fall1 Russell Johnsen1 Kaite Honeyman1 Pat Iversen2 Susan Fletcher1 and Stephen D Wilton 1 Address Experimental Molecular Medicine Group Centre for Neuromuscular and Neurological Disorders University of Western Australia Nedlands Perth 6009 Western Australia and 2AVI BioPharma Corvallis Oregon USA Email Abbie M Fall - afall@ Russell Johnsen - rjohnsen@ Kaite Honeyman - khoney@ Pat Iversen - piversen@ Susan Fletcher - sfletch@ Stephen D Wilton - swilton@ Corresponding author Published 24 May 2006 Received 01 February 2006 Genetic Vaccines and Therapy 2006 4 3 doi 186 1479-0556-4-3 Accepted 24 May 2006 This article is available from http content 4 l 3 2006 Fall 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 Duchenne muscular dystrophy is a fatal genetic disorder caused by dystrophin gene mutations that result in premature termination of translation and the absence of functional protein. Despite the primary dystrophin gene lesion immunostaining studies have shown that at least 50 of DMD patients mdx mice and a canine model of DMD have rare dystrophin-positive or revertant fibres. Fine epitope mapping has shown that the majority of transcripts responsible for revertant fibres exclude multiple exons one of which includes the dystrophin mutation. Methods The mdx mouse model of muscular dystrophy has a nonsense mutation in exon 23 of the dystrophin gene. We have shown that antisense oligonucleotides AOs can induce the removal of .