Results 81 to 90 of about 641,320 (378)

Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial Dysautonomia

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Familial dysautonomia (FD) is a hereditary neurodevelopmental disorder caused by aberrant splicing of the ELP1 gene, leading to a tissue‐specific reduction in ELP1 protein expression. Preclinical models indicate that increasing ELP1 levels can mitigate disease manifestations.
Alejandra González‐Duarte, Lucy Norcliffe‐Kaufmann, Maria Luisa Cotrina, Zenith Khan, Kaia Dalamo, Patricio Millar Vernetti, Matthew Lawless, Elisabetta Morini, Monica Salani, Marla Weetall, Jana Narasimhan, Agostino G. Rocha, Susan A. Slaugenhaupt, Horacio Kaufmann   +13 more
wiley   +1 more source

2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA

Molecular Therapy: Nucleic Acids, 2020
Small interfering RNAs (siRNAs) have the potential to treat a broad range of diseases. siRNAs need to be extensively chemically modified to improve their bioavailability, safety, and stability in vivo.
Sarah M. Davis, Jacquelyn Sousa, Lorenc Vangjeli, Matthew R. Hassler, Dimas Echeverria, Emily Knox, Anton A. Turanov, Julia F. Alterman, Anastasia Khvorova   +8 more
doaj   +1 more source

Olink Proteomics Analysis Reveals Heterogeneous Responses to FcRn Blockade in Anti‐AChR Antibody‐Positive Myasthenia Gravis: FGF‐19 as a Novel Biomarker

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective This study aimed to systematically observe the clinical manifestations, immune cell subsets, and dynamic changes in serological indicators in patients with myasthenia gravis (MG) before and after efgartigimod (EFG) treatment. Methods We analyzed the baseline data, laboratory parameters, and lymphocyte subset proportions in MG ...
Tiancheng Luo, Deyou Peng, Zhouao Zhang, Mingjin Yang, Xinyan Guo, Tianyu Ma, Xiaoyu Huang, Mingming Xu, Linlin Fu, Yong Zhang   +9 more
wiley   +1 more source

A Polymerase-chain-reaction Assay for the Specific Identification of Transcripts Encoded by Individual Carcinoembryonic Antigen (CEA)-gene-family Members [PDF]

, 1993
Carcinoembryonic antigen (CEA) is a tumor marker that belongs to a family of closely related molecules with variable expression patterns. We have developed sets of oligonucleotide primers for the specific amplification of transcripts from individual CEA ...
Ang, Berling, Bernards, Chi, Chomczynski, Chomczynski, Daniel, F. Kommoss, Fainstein, Hinoda, Hinoda, J. Thompson, Khan, Kim, Kuroki, Lathe, Lucas, Motoyama, N. Beauchemin, Nagel, Neumaier, Ng, Oikawa, Ponte, Pèlegrin, Rooney, S. Mössinger, S. von Kleist, Sakuragi, Sambrook, Shively, Thompson, U. Engels, V. Reichardt, W. Zimmermann   +34 more
core   +1 more source

Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care.
Amin Mehrabian, Peter Auguste, Amy Grove, Anna Brown, Janette Parr, Mubarak Patel, Furqan Butt, Jeremiah Donoghue, Mehdi Yousefi, Jo Parsons   +9 more
wiley   +1 more source

A Visual Compendium of Principal Modifications within the Nucleic Acid Sugar Phosphate Backbone

Molecules
Nucleic acid chemistry is a huge research area that has received new impetus due to the recent explosive success of oligonucleotide therapy. In order for an oligonucleotide to become clinically effective, its monomeric parts are subjected to ...
Daria Novikova, Aleksandra Sagaidak, Svetlana Vorona, Vyacheslav Tribulovich   +3 more
doaj   +1 more source

Application of COMPOCHIP Microarray to Investigate the Bacterial Communities of Different Composts [PDF]

, 2009
A microarray spotted with 369 different 16S rRNA gene probes specific to microorganisms involved in the degradation process of organic waste during composting was developed. The microarray was tested with pure cultures, and of the 30,258 individual probe-
A Alfreider, A Loy, A Loy, BEE Buchholz-Cleven, Brigitte A. Knapp, CJF Braak ter, D Guschin, D-Y Lee, DA Stahl, DM Barrett, EW Alm, F Schwieger, F Wintzingerode von, G Muyzer, G Taroncher-Oldenburg, G Varani, H Daims, H Sanguin, H Urakawa, HAJ Hoitink, HAJ Hoitink, HAJ Hoitink, Heribert Insam, HM Dionisi, IH Franke-Whittle, IH Franke-Whittle, Ingrid H. Franke-Whittle, J Peplies, J Ryckeboer, Jacques Fuchs, JG Fuchs, K Nakasaki, K-H Kim, KJ Breslauer, L Bodrossy, L Bodrossy, L Ludwig, M Blanc, M Danon, M Hogardt, MA Elorrieta, MT Suzuki, N Stralis-Pavese, P Legendre, P Schloss, PM Dees, R Amann, Ruediger Kaufmann, S Haruta, SF Altschul, SM Tiquia, SM Tiquia, T Beffa, W-T Liu, Y Koizumi   +54 more
core   +1 more source

Characterization of Clinical Phenotype to Glial Fibrillary Acidic Protein Concentrations in Alexander Disease

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To determine the concentration of glial fibrillary acidic protein (GFAP) in cerebrospinal fluid (CSF) and plasma in Alexander disease (AxD) and whether GFAP levels are predictive of disease phenotypes. Methods CSF and plasma were collected (longitudinally when available) from AxD participants and non‐AxD controls.
Amy T. Waldman, Asako Takanohashi, Joshua Y. Joung, Geraldine W. Liu, Kaley Arnold, Amy Pizzino, Walter Faig, Sarah Woidill, Sona Narula, Adeline L. Vanderver   +9 more
wiley   +1 more source

probeBase—an online resource for rRNA-targeted oligonucleotide probes and primers: new features 2016

Nucleic Acids Res., 2015
probeBase http://www.probebase.net is a manually maintained and curated database of rRNA-targeted oligonucleotide probes and primers. Contextual information and multiple options for evaluating in silico hybridization performance against the most recent ...
D. Greuter, A. Loy, M. Horn, T. Rattei
semanticscholar   +1 more source

Oligonucleotides

, 2019
Oligonucleotides are short chains of single stranded or double stranded nucleotides, either DNA, RNA or a combination and in some cases chemically modified to improve their application as a drug substance. They are applied to modulate endogenous mechanisms of DNA and RNA processing, thereby modifying disease pathways at the genetic level, rather than ...
Schiffelers, Raymond M., Oude Blenke, Erik, Mastrobattista, Enrico   +2 more
openaire   +2 more sources