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

极深

扩张

Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophythat 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

带来墨水

Inotersen for the Treatment of Hereditary Transthyretin Amyloidosiss of the liver cells. By doing so, it prevents the production of the mutant and wild-type forms of transthyretin, impeding the progression of the disease. In this article, the mechanism of action and safety profile of inotersen will be discussed along with some future directions following its approv

separate

misshapen

Albumin-Binding Fatty Acid–Modified Gapmer Antisense Oligonucleotides for Modulation of Pharmacokineothioate 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.

礼节

CHAFE

Invention and Early History of Gapmers sequence complementarity, gapmers recruit ribonuclease H and induce target RNA degradation. Since its concept first emerged in the 1980s, much work has gone into developing gapmers for use in basic research and therapy. These include improvements in gapmer chemistry, delivery, and therapeutic safet

评论性

案发地点

follicular-unit

Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington’s Diseaseogenicity, thereby providing considerable insight to develop suitable therapies. With the successful translation of antisense oligonucleotides (AOs) from in vitro into animal models and clinical practice, modifications are being continuously made to the AOs to improve the pharmacokinetics and pharma