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Sequence-Specific DNA Binders for the Therapy of Mitochondrial Diseases (Paperback, 1st ed. 2022)
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Sequence-Specific DNA Binders for the Therapy of Mitochondrial Diseases (Paperback, 1st ed. 2022)
Series: Springer Theses
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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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