New Gene Therapy Strategies for the Deletion of Exon 44 of Dystrophin Gene Based on Gene Editing by TALENs
New Gene Therapy Strategies for the Deletion of Exon 44 of Dystrophin Gene Based on Gene Editing by TALENs作者机构：Centre Hospitalier de l’Université Laval Research Center Laval University Quebec Canada Department of Molecular Diagnostics and Biopharmaceutics College of Pharmaceutical Science Soochow University Suzhou China DNA Sequencing Core Laboratory City of Hope National Medical Center Duarte USA Gene Core The Genomics Institute of the Novartis Research Foundation San Diego USA University of Waterloo Waterloo Canada
出 版 物：《Open Journal of Medicinal Chemistry》 (药物化学期刊（英文）)
年 卷 期：2013年第3卷第1期
页 面：1-6页
学科分类：1002[医学-临床医学] 100214[医学-肿瘤学] 10[医学]
主 题：TALENs DMD Gene Therapy Exon Skipping
摘 要：Duchenne Muscular Dystrophy (DMD) is a severe childhood form of muscular dystrophy. Both the severe form and its milder form of Becker Muscular Dystrophy (BMD) are caused by the mutation of dystrophin gene. Different from some other genetic diseases such as hemophilia that can be treated by replacement therapy, there is no effective therapy for muscular dystrophy in conventional medication. Gene editing technology from the recently developed engineered nucleases such as TALENs has been successfully employed in genome modification of a variety of species, and will be applied in gene therapy of selected human diseases. The genetic basis of DMD and BMD indicates that DMD is a good target for gene therapy through returning the reading frame of dystrophin gene. Gene therapy strategies described here may apply to many other genetic diseases. Wider application of TALENs in gene therapy have the potential to dramatically prolong the lifespan of individuals with genetic diseases.
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