Titlebook: Gapmers; Methods and Protocol Toshifumi Yokota,Rika Maruyama Book 2020 Springer Science+Business Media, LLC, part of Springer Nature 2020 A

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A Generalization of the Fibonacci Search,ation for the treatment of familial hypercholesterolemia and transthyretin amyloidosis-associated polyneuropathy. Here, we review the events surrounding the early development of gapmers, from conception to their maturity, and briefly conclude with perspectives on their use in therapy.

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Evaluating the Knockdown Activity of MALAT1 ENA Gapmers In Vitrouse it does not involve the use of any transfection reagent and has minimal effects on cell growth. Further, we describe a convenient technique for performing one-step reverse transcription and real-time qPCR using cell lysates without RNA extraction. Data for up to 96 samples can be obtained following these methods.

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Interdisciplinary Systems Researchvo than the parent ASO. Thereafter the HDO strategy was found to be also effective for improving the properties of ASOs modulating blood–brain barrier function and ASO antimiRs which are RNase H-independent ASOs. Therefore, the HDO strategy has been shown to be versatile technology platform to develop effective nucleic acid drugs.

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Piergiulio Corsini,Violeta Leoreanued. The methods comprise the design of gapmer ASOs and the in vitro evaluation of gapmer ASOs on the specific silencing of the mutant allele at mRNA levels, and functional assessment at protein levels. A fibroblast cell line cultured from a UCMD patient carrying a dominant mutation in one of the . genes is used as a cellular model.

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