书目名称 | Sequence-Specific DNA Binders for the Therapy of Mitochondrial Diseases | 编辑 | Takuya Hidaka | 视频video | | 概述 | Is nominated as an outstanding Ph.D. thesis by Kyoto University.Provides a study for a transgene-free gene therapy for mitochondrial diseases.Describes DNA-binding molecules to control nuclear or mito | 丛书名称 | Springer Theses | 图书封面 |  | 描述 | This book describes the author’s work on the development of sequence-specific DNA binders for the therapy of mitochondrial diseases. In the first chapter, the author provides a detailed background of pyrrole–imidazole polyamides (PIPs) and mitochondrial disease research followed by chapters presenting the author‘s own research and discoveries. Firstly, the developed compounds called MITO-PIPs, which recognize specific sequences in mitochondrial DNA, are presented. The following chapter demonstrates how, by introducing a DNA alkylating reagent into a MITO-PIP that recognizes the adjacent sequence to a target mutation, the copy number of mutated mitochondrial DNA was successfully reduced in live cells. Furthermore, because nuclear DNA is another important target for treating mitochondrial diseases, the author demonstrated that tri-arginine vectors can enhance nuclear uptake of PIPs and improve their biological activity in cells..This work willattract readers’ interest because it paves the way for a transgene-free chemical gene therapy for mitochondrial diseases. The book includes a detailed description of experimental procedures, especially compound synthesis. This description helps | 出版日期 | Book 2022 | 关键词 | Mitochondrial diseases; Pyrrole–imidazole polyamides; DNA-binding molecules; DNA mutation; DNA alkylatio | 版次 | 1 | doi | https://doi.org/10.1007/978-981-16-8436-4 | isbn_softcover | 978-981-16-8438-8 | isbn_ebook | 978-981-16-8436-4Series ISSN 2190-5053 Series E-ISSN 2190-5061 | issn_series | 2190-5053 | copyright | Springer Nature Singapore Pte Ltd. 2022 |
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