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Sequence-specific DNA binders for the therapy of mitochondrial diseases

紀錄類型:	書目-電子資源 : Monograph/item
正題名/作者:	Sequence-specific DNA binders for the therapy of mitochondrial diseasesby Takuya Hidaka.
作者:	Hidaka, Takuya.
出版者:	Singapore :Springer Singapore :2022.
面頁冊數:	xiii, 94 p. :ill., digital ;24 cm.
Contained By:	Springer Nature eBook
標題:	Mitochondrial pathologyGenetic aspects.
電子資源:	https://doi.org/10.1007/978-981-16-8436-4
ISBN:	9789811684364$q(electronic bk.)

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

Sequence-specific DNA binders for the therapy of mitochondrial diseases[electronic resource] /by Takuya Hidaka. - Singapore :Springer Singapore :2022. - xiii, 94 p. :ill., digital ;24 cm. - Springer theses,2190-5061. - Springer theses..

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 Pyrrole-Imidazole Polyamides by Incorporation of the Tri-arginine Vector.

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 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 author's studies and to perform similar and extended studies themselves.

ISBN: 9789811684364$q(electronic bk.)

Standard No.: 10.1007/978-981-16-8436-4doiSubjects--Topical Terms:

918912
Mitochondrial pathology
--Genetic aspects.

LC Class. No.: RB147.5 / .H53 2022

Dewey Class. No.: 616.042

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520 $a 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 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 author's studies and to perform similar and extended studies themselves.
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