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

Hidaka, Takuya, author.
2022
RB147.5 .H53 2022
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Title
Sequence-specific DNA binders for the therapy of mitochondrial diseases / Takuya Hidaka.
Author
Hidaka, Takuya, author.
ISBN
9789811684364 (electronic bk.)
9811684367 (electronic bk.)
9789811684357
9811684359
DOI
https://doi.org/10.1007/978-981-16-8436-4
Published
Singapore : Springer, [2022]
Copyright
©2022
Language
English
Description
1 online resource (104 pages) : illustrations (chiefly color).
Item Number
10.1007/978-981-16-8436-4 doi
Call Number
RB147.5 .H53 2022
Dewey Decimal Classification
616/.042
Summary
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.
Note
"Doctoral thesis accepted by Kyoto University, Kyoto, Japan."
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Includes bibliographical references.
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Access limited to authorized users.
Source of Description
Description based upon print version of record.
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Springer theses.
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Sequence-Specific DNA Binders for the Therapy of Mitochondrial Diseases
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General Introduction
Creation of Synthetic Ligand for Mitochondrial DNA sequence Recognition and Promoter-Specific Transcription Suppression
Allele-Specific Replication Inhibition of Mitochondrial DNA by MITO-PIP Conjugated with Alkylation Reagent
Enhanced Nuclear Accumulation of PyrroleImidazole Polyamides by Incorporation of the Tri-arginine Vector.

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