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Sequence-specific DNA binders for the therapy of mitochondrial diseases / Takuya Hidaka.

9789811684364 (electronic bk.)
9811684367 (electronic bk.)
9789811684357
9811684359

Singapore : Springer, [2022]

©2022

English

1 online resource (104 pages) : illustrations (chiefly color).

10.1007/978-981-16-8436-4 doi

RB147.5 .H53 2022

616/.042

This book describes the authors 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 pyrroleimidazole 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 will attract 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 readers to have a clear image of the authors studies and to perform similar and extended studies themselves.

"Doctoral thesis accepted by Kyoto University, Kyoto, Japan."

Includes bibliographical references.

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Description based upon print version of record.

Springer theses.

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