001443611 000__ 03879cam\a2200601Ii\4500 001443611 001__ 1443611 001443611 003__ OCoLC 001443611 005__ 20230310003553.0 001443611 006__ m\\\\\o\\d\\\\\\\\ 001443611 007__ cr\cn\nnnunnun 001443611 008__ 220108s2022\\\\si\a\\\\ob\\\\000\0\eng\d 001443611 019__ $$a1290840699$$a1291147238$$a1291171697 001443611 020__ $$a9789811684364$$q(electronic bk.) 001443611 020__ $$a9811684367$$q(electronic bk.) 001443611 020__ $$z9789811684357 001443611 020__ $$z9811684359 001443611 0247_ $$a10.1007/978-981-16-8436-4$$2doi 001443611 035__ $$aSP(OCoLC)1291317724 001443611 040__ $$aEBLCP$$beng$$erda$$epn$$cEBLCP$$dYDX$$dGW5XE$$dOCLCO$$dOCLCF$$dOCLCO$$dUKAHL$$dOCLCQ 001443611 049__ $$aISEA 001443611 050_4 $$aRB147.5$$b.H53 2022 001443611 08204 $$a616/.042$$223 001443611 1001_ $$aHidaka, Takuya,$$eauthor. 001443611 24510 $$aSequence-specific DNA binders for the therapy of mitochondrial diseases /$$cTakuya Hidaka. 001443611 264_1 $$aSingapore :$$bSpringer,$$c[2022] 001443611 264_4 $$c©2022 001443611 300__ $$a1 online resource (104 pages) :$$billustrations (chiefly color). 001443611 336__ $$atext$$btxt$$2rdacontent 001443611 337__ $$acomputer$$bc$$2rdamedia 001443611 338__ $$aonline resource$$bcr$$2rdacarrier 001443611 4901_ $$aSpringer theses 001443611 500__ $$a"Doctoral thesis accepted by Kyoto University, Kyoto, Japan." 001443611 504__ $$aIncludes bibliographical references. 001443611 5050_ $$aGeneral 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. 001443611 506__ $$aAccess limited to authorized users. 001443611 520__ $$aThis 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. 001443611 588__ $$aDescription based upon print version of record. 001443611 650_0 $$aMitochondrial pathology$$xGenetic aspects. 001443611 650_0 $$aMitochondrial DNA$$xAbnormalities. 001443611 650_0 $$aPolyamides. 001443611 650_0 $$aGene therapy. 001443611 650_6 $$aMaladies mitochondriales$$xAspect génétique. 001443611 650_6 $$aPolyamides. 001443611 650_6 $$aThérapie génique. 001443611 655_0 $$aElectronic books. 001443611 77608 $$iPrint version:$$aHidaka, Takuya$$tSequence-Specific DNA Binders for the Therapy of Mitochondrial Diseases$$dSingapore : Springer Singapore Pte. Limited,c2022$$z9789811684357 001443611 830_0 $$aSpringer theses. 001443611 852__ $$bebk 001443611 85640 $$3Springer Nature$$uhttps://univsouthin.idm.oclc.org/login?url=https://link.springer.com/10.1007/978-981-16-8436-4$$zOnline Access$$91397441.1 001443611 909CO $$ooai:library.usi.edu:1443611$$pGLOBAL_SET 001443611 980__ $$aBIB 001443611 980__ $$aEBOOK 001443611 982__ $$aEbook 001443611 983__ $$aOnline 001443611 994__ $$a92$$bISE