Results 231 to 240 of about 125,197 (343)

Synthesis and evaluation of (S)-5′-C-aminopropyl and (S)-5′-C-aminopropyl-2′-arabinofluoro modified DNA oligomers for novel RNase H-dependent antisense oligonucleotides

, 2020
Yujun Zhou, Ryohei Kajino, Seiichiro Ishii, Kenji Yamagishi, Yoshihito Ueno   +4 more
openalex   +2 more sources

Olezarsen in Hypertriglyceridemia With High Cardiac Risk: A GRADE‐Assessed Meta‐Analysis of Randomized Trials With Trial Sequential Evidence

Endocrinology, Diabetes &Metabolism, Volume 9, Issue 3, May 2026.
This meta‐analysis of randomized trials evaluates olezarsen for hypertriglyceridemia in patients at high cardiovascular risk. Olezarsen significantly improved triglycerides and other atherogenic lipid parameters with generally acceptable safety, supporting its potential as a promising adjunct therapy while highlighting the need for larger and longer ...
Ahmed Emara, Ameer Awashra, Heba Aboeldahab, Ahmed Farid Gadelmawla, Hamza A. Abdul‐Hafez, Abdullah M. Alharran, Amal A. Alsubaiei, Muneera Jasim AlRumaihi, Sara Ahmed Albuhmaid, Abdalhakim Shubietah   +9 more
wiley   +1 more source

The Ubiquitin Ligase Zinc Finger SWIM Domain‐Containing Protein 8 Regulates Oligodendrocyte Development Through the Argonaute2/MicroRNA‐7 Axis

Glia, Volume 74, Issue 5, May 2026.
ZSWIM8, an E3 ubiquitin ligase that broadly targets IDR‐rich proteins, drives MiR‐7‐dependent AGO2 degradation and the turnover of many RBPs. Loss of ZSWIM8 leads to MiR‐7 accumulation in oligodendrocyte progenitor cells and myelination defects in the developing brain. ABSTRACT Proteostasis of proteins with intrinsically disordered regions (IDRs) is of
Jing Lei, Siming Zhong, Rong Fan, Xin Shu, Guan Wang, Jiansheng Guo, Shuting Xue, Luqian Zheng, Aiming Ren, Junfang Ji, Bing Yang, Shumin Duan, Zhiping Wang, Xing Guo   +13 more
wiley   +1 more source

OpenASO: RNA Rescue-designing splice-modulating antisense oligonucleotides through community science. [PDF]

RNA
Tse V, Guiterrez M, Townley J, Romano J, Pearl J, Pearl J, Chacaltana G, Players E, Das R, Sanford JR, Stone MD.   +10 more
europepmc   +1 more source

A Cationic Block Co‐Polymer for Gene Delivery in the Posterior Segment of the Eye

Journal of Biomedical Materials Research Part A, Volume 114, Issue 5, May 2026.
ABSTRACT Diseases of the back of the eye such as neovascular age‐related macular degeneration (nAMD) are vision threatening and treatment is burdensome for patients, often requiring ocular injections every other month. Injection risks and logistics lower patient compliance; however, even patients receiving optimal treatment can deteriorate.
Amber Monteiro, Odunayo O. Mugisho, Antonio de Souza, Lina Liu, Yosra Agban, Priyanka Agarwal, Avik Shome, Ilva D. Rupenthal, Heather Sheardown   +8 more
wiley   +1 more source

Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system. [PDF]

Nucleic Acids Res
O'Rourke JG, Bachmann G, Mazur C, Zhou K, Platoshyn O, Bravo Hernandez M, Klein SK, Nguyen J, Burel S, Hoffmaster C, Zanardi T, Jafar-Nejad P, Marsala M, Henry SP, Swayze EE, Powers B, Kordasiewicz HB.   +16 more
europepmc   +1 more source

Offline comprehensive liquid chromatography in combination with a Deoxyribonuclease I immobilized enzymatic reactor for selective screening of oligonucleotide mixtures [PDF]

, 2016
Alvarez Porebski, Piotr Wiktor, Lynen, Frederic   +1 more
core   +1 more source

2025 Consensus Clinical Management Guidelines for Niemann‐Pick Disease Type C

Journal of Inherited Metabolic Disease, Volume 49, Issue 3, May 2026.
ABSTRACT In 2018, the International Niemann‐Pick Disease Alliance (INPDA) and the International Niemann‐Pick Disease Registry (INPDR) developed and published comprehensive clinical management guidelines to support inclusive and standardized care pathways in Niemann‐Pick disease type C (NPC)—an ultra‐rare, autosomal recessive, neurovisceral lysosomal ...
Tarekegn Hiwot, Forbes D. Porter, Tatiana Bremova‐Ertl, Uma Ramaswami, Caroline Hastings, Bénédicte Héron, Justin Hopkin, Joella Melville, Hernan Amartino, Mireia del Toro, Federica Deodato, Fatih Ezgü, Paul Gissen, James B. Gibson, Can Ficicioglu, Roberto Giugliani, Orna Staretz‐Chacham, Frances Platt, Nathalie Guffon, Kristina Julich, Nikola Kresojević, Anna Lehman, Yann Nadjar, Susanne A. Schneider, Simon Jones, Eugen Mengel, Michel Tchan, Mark Walterfang, Ozlem Goker‐Alpan, Charlotte Dawson, Sandra Cowie, Toni Mathieson, Elizabeth Berry‐Kravis, Marc C. Patterson   +33 more
wiley   +1 more source

ASOptimizer: optimizing chemical diversity of antisense oligonucleotides through deep learning. [PDF]

Nucleic Acids Res
Kang S, Lee D, Hwang G, Lee K, Kang M.
europepmc   +1 more source

Unifying the Communities of Early‐Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study of GBE1‐Related Disease

JIMD Reports, Volume 67, Issue 3, May 2026.
Genetic prevalence study of glycogen storage disease type IV. In collaboration with the Rare Genomes Project at the Broad Institute of MIT and Harvard and the APBD Research Foundation, this study queried and curated variants in GBE1 from ClinVar, HGMD, and gnomAD to calculate the genetic prevalence of glycogen storage disease type IV (GSD IV).
Rebecca L. Koch, H. Orhan Akman, Erin Chown, Deberah Goldman, Jeff Levenson, Qing Lu, Lindsay T. Michalovicz Gill, Matthew Morgan, Jennifer L. Orthmann‐Murphy, Natacha T. Pires, Rebecca Reef, Harriet Saxe, Moriel Singer‐Berk, Samantha Baxter   +13 more
wiley   +1 more source