书目名称 | Muscle Gene Therapy | 副标题 | Methods and Protocol | 编辑 | Dongsheng Duan | 视频video | http://file.papertrans.cn/642/641406/641406.mp4 | 概述 | Presents a comprehensive collection of state-of-the-art muscle gene therapy protocols from leaders in the field.Focuses on gene delivery in less-covered large animal models, such as swine, ovine, cani | 丛书名称 | Methods in Molecular Biology | 图书封面 |  | 描述 | Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body.In Muscle Gene Therapy: Methods and Protocols, expert researchers in the field present a collection of techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent rodent models and vitally important larger animal models.Divided into three sections, this volume examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle gene therapy technology such as adeno-associated viral vector (AAV), oligonucleotide-mediated exon-skipping, and novel RNA-based strategies, and step-by-step guidance on muscle gene delivery in swine, ovine, canine, and non-human primates.Written in the highly successful Methods in Molecular Biology™ series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, detailed, readily reproducible laboratory protocols, and tips on troublesh | 出版日期 | Book 2011 | 关键词 | AAV; Adeno-associated viral vectors; Delivery systems; Exon-skipping; Gene expression cassette; Gene muta | 版次 | 1 | doi | https://doi.org/10.1007/978-1-61737-982-6 | isbn_softcover | 978-1-4939-6182-5 | isbn_ebook | 978-1-61737-982-6Series ISSN 1064-3745 Series E-ISSN 1940-6029 | issn_series | 1064-3745 | copyright | Springer Science+Business Media, LLC 2011 |
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