Sequence-Specific DNA Binders for the Therapy of Mitochondrial Diseases



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Éditeur :

Springer


Collection :

Springer Theses

Paru le : 2022-01-03



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Description




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 readers to have a clear image of the author’s studies and to perform similar and extended studies themselves.

Pages
94 pages
Collection
Springer Theses
Parution
2022-01-03
Marque
Springer
EAN papier
9789811684357
EAN PDF
9789811684364

Informations sur l'ebook
Nombre pages copiables
0
Nombre pages imprimables
9
Taille du fichier
4495 Ko
Prix
147,69 €
EAN EPUB
9789811684364

Informations sur l'ebook
Nombre pages copiables
0
Nombre pages imprimables
9
Taille du fichier
27392 Ko
Prix
147,69 €