Описание: 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.
Описание: 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.
Автор: Justin C. St. John Название: Mitochondrial DNA, Mitochondria, Disease and Stem Cells ISBN: 1627038671 ISBN-13(EAN): 9781627038676 Издательство: Springer Рейтинг: Цена: 22201.00 р. Наличие на складе: Есть у поставщика Поставка под заказ.
Описание: The reader is then introduced to how mtDNA is transmitted through the oocyte and how stem cells can be used to study mitochondrial biogenesis and mtDNA replication and transcription in undifferentiated pluripotent and differentiating cells and how mitochondria adapt during this process.
Автор: Navas Название: Mitochondrial Diseases ISBN: 3030701492 ISBN-13(EAN): 9783030701499 Издательство: Springer Рейтинг: Цена: 19564.00 р. Наличие на складе: Есть у поставщика Поставка под заказ.
Описание: Mitochondrial diseases comprise a clinically and genetically heterogeneous group of rare disorders that may affect virtually any system of the body at any age. Due to their complexity, understanding and diagnosing these diseases requires a multidisciplinary approach. This book provides an update on the major features of human mitochondrial diseases: genetic bases, pathophysiology, diagnosis, and treatment, and of the new technologies involved in the diagnosis and on the characterization of patients. The 11 chapters examine the unique complex interactions between the mitochondrial and the nuclear genomes involved in the biogenesis and the regulation of the mitochondrial respiratory chain, and their relevance to human disease. We discuss the traditional biochemical and genetic approaches, as well as the new omic technologies, and the cellular and animal models used in mitochondrial research. The last chapter is dedicated to the current treatment options. Authors are worldwide experts in these fields and integrate expertise in both basic science and clinical research. This book is particularly important for both scientists and clinicians interested in the diagnosis and treatment of these diseases.
Автор: Navas Placido, Salviati Leonardo Название: Mitochondrial Diseases: Theory, Diagnosis and Therapy ISBN: 3030701468 ISBN-13(EAN): 9783030701468 Издательство: Springer Цена: 19564.00 р. Наличие на складе: Есть у поставщика Поставка под заказ.
Описание: PrefaceChapter 1 Two genomes connection: biogenesis of mitochondria respiratory chain Chapter 2 Nuclear DNA: mutations in structural subunits and assembly factorsChapter 3 Defects of mtDNAChapter 4 MtDNA maintenance genes and depletionChapter 5 Defects in mitochondrial protein synthesisChapter 6 Defects in mitochondria dynamicsChapter 7 CoQ biosynthesis disordersChapter 8 Cytochrome c mutationsChapter 9 Biochemical approaches for mitochondrial diseases diagnosisChapter 10 Molecular genetics in the Next Generation Sequencing era Chapter 11 Model cells and organismsChapter 12 Metabolomics and proteomics: complementary toolsChapter 13 Therapies approaches in mitochondrial diseasesIndex
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