Results 141 to 150 of about 275,477 (365)

Longitudinal Analysis of Neuroradiological Biomarkers for Fragile X‐Associated Tremor/Ataxia Syndrome and Implications for Clinical Trials

Annals of Neurology, EarlyView.
Objective The objective of this study was to show the capacity of structural brain magnetic resonance imaging (MRI) measures to serve as monitoring biomarkers for Fragile X‐Associated Tremor/Ataxia Syndrome (FXTAS). Methods From 2 longitudinal studies of male FMR1 premutation carriers, 2 brain MRI scans were selected from each participant, collected ...
David Hessl, Jun Yi Wang, Glenda Espinal, Ellery Santos, Flora Tassone, Randi J. Hagerman, Susan M. Rivera   +6 more
wiley   +1 more source

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice

Nucleic Acids Research, 2014
Triantennary N-acetyl galactosamine (GalNAc, GN3), a high-affinity ligand for the hepatocyte-specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation gapmer antisense oligonucleotides (ASOs) 6–10-fold in mouse liver.
T. P. Prakash, M. Graham, Jinghua Yu, Rick Carty, Audrey Low, A. Chappell, Karsten Schmidt, Chenguang Zhao, M. Aghajan, Heather F Murray, Stan J. Riney, S. Booten, S. Murray, H. Gaus, J. Crosby, W. Lima, Shuling Guo, B. Monia, E. Swayze, P. Seth   +19 more
semanticscholar   +1 more source

Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies

Annals of Neurology, EarlyView.
Autosomal recessive cerebellar ataxias are disabling neurodegenerative genetic conditions affecting balance and coordination. Advancements in genomic testing have improved diagnosis, leading to a new focus on the development of targeted precision therapeutics addressing cellular, biochemical, and genetic disease mechanisms with a resulting emphasis on ...
Brent L. Fogel, Thomas Klopstock, David R. Lynch, Francesca Maltecca, Mayank Verma, Berge A. Minassian, Frances M. Platt, Débora Farina Gonçalves, Hélène Puccio, Andreas Roos, Matthis Synofzik   +10 more
wiley   +1 more source

Investigating discovery strategies and pharmacological properties of stereodefined phosphorodithioate LNA gapmers

Molecular Therapy: Nucleic Acids, 2022
The introduction of sulfur into the phosphate linkage of chemically synthesized oligonucleotides creates the stereocenters on phosphorus atoms. Researchers have valued the nature of backbone stereochemistry and early on investigated drug properties for ...
Jörg Duschmalé, Adrian Schäublin, Erik Funder, Steffen Schmidt, Łukasz J. Kiełpiński, Helle Nymark, Klaus Jensen, Troels Koch, Martina Duschmalé, Erich Koller, Marianne Ravn Møller, Simone Schadt, Christophe Husser, Andreas Brink, Sabine Sewing, Tanja Minz, Jesper Wengel, Konrad Bleicher, Meiling Li   +18 more
doaj  

Suppression of kinesin expression in cultured hippocampal neurons using antisense oligonucleotides. [PDF]

, 1992
Adriana Ferreira, J Niclas, Ron Vale, Gary Banker, Kenneth S. Kosik   +4 more
openalex   +1 more source

Association of antisense oligonucleotides with lipoproteins prolongs the plasma half-life and modifies the tissue distribution [PDF]

, 1991
P.C. de Smidt, Trung Le Doan, Sandro De Falco, Theo J.C. van Berkel   +3 more
openalex   +1 more source

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

Nucleic Acids Research, 2014
Fully phosphorothioate antisense oligonucleotides (ASOs) with locked nucleic acids (LNAs) improve target affinity, RNase H activation and stability. LNA modified ASOs can cause hepatotoxicity, and this risk is currently not fully understood.
Andrew D. Burdick, Simone Sciabola, Srinivasa R. Mantena, B. Hollingshead, R. Stanton, J. Warneke, Mingyue Zeng, E. Martsen, A. Medvedev, S. Makarov, Lori A Reed, J. W. Davis, L. Whiteley   +12 more
semanticscholar   +1 more source

Noncoding RNAs in Host–Microbiota Interaction

Animal Research and One Health, EarlyView.
The intricate interplay between gut microbiota and the host exerts a profound impact on host biological processes and disease development. This review highlights the pivotal role of noncoding RNAs (ncRNAs) in this interaction. Host ncRNAs regulate gene expression and microbiota composition, whereas microbial small RNAs (sRNAs) modulate host gene ...
Ziyi Zhao, Ni Li, Jingyi Xu, Jianrong Ren, Xiaojun Yang, Xinyi Li, Junhu Yao, Shengru Wu   +7 more
wiley   +1 more source

Antisense oligonucleotides in solution or encapsulated in immunoliposomes inhibit replication of HIV-1 by several different mechanisms [PDF]

, 1994
Olivier Zelphati, Jean‐Louis Imbach, Nathalie Signoret, Gerald Zon, Bernard Rayner, Lee Leserman   +5 more
openalex   +1 more source

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

British Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić, Elvira Meni Maria Gkrinia, Patrick Eustaquio, Andrea Katrin Faour, Dinko Vitezić   +4 more
wiley   +1 more source