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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1064-3745 tocols, and tips on troubleshooting and avoiding known pitfalls... Authoritative and cutting-edge, .Gapmers: Methods and Protocols. aims to provide diverse applications of gapmers along with protocols that will assist readers in moving the frontier..978-1-0716-0773-2978-1-0716-0771-8Series ISSN 1064-3745 Series E-ISSN 1940-6029

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https://doi.org/10.1007/978-94-009-0223-7that have a PS backbone flanked with the modified AOs on both sides. Mipomersen (trade name Kynamro), a 2′-.-methoxyethyl (MOE) gapmer, was approved by the Food and Drug Administration (FDA) for the treatment of homozygous familial hypercholesterolemia (HoFH) in 2013. Volanesorsen, another 20-mer MO

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Computing with the Unpredictable RNase H cleavage while having enhanced target binding affinity and nuclease resistance. This chapter will consolidate the different strategies studied thus far to develop a treatment for DM1 through the targeting of toxic repetitive RNA using gapmers.

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Geometric and Algebraic Canonical Formsothioate or phosphodiester backbone modifications. This work offers a strategy to optimize gapmer ASO pharmacokinetics by a proposed endogenous assembly process with serum albumin that can be tuned by gapmer ASO design modifications.

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Jumpei Tsujiuchi,George W. Strokef72 repeat expanded transcripts resulted in recovery from the disease-related phenotypes in patient-derived fibroblasts. Our findings highlight the therapeutic potential of C9ALS using this gapmer oligonucleotide-based approach.

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