Results 71 to 80 of about 635,743 (331)

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

Linear model for fast background subtraction in oligonucleotide microarrays [PDF]

, 2009
One important preprocessing step in the analysis of microarray data is background subtraction. In high-density oligonucleotide arrays this is recognized as a crucial step for the global performance of the data analysis from raw intensities to expression ...
Barkema, Gerard T., Carlon, Enrico, Kroll, K. Myriam   +2 more
core   +4 more sources

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

Regulation of metabolism by miR-378 [PDF]

, 2014
The present invention provides a method of regulating fatty acid metabolism in a cell by contacting the cell with a modulator of miR-378 and/or miR-378* activity or expression.
Carrer, Michele, Olson, Eric N.
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

Primary Human Tissue Models for Metabolic Dysfunction‐Associated Liver Disease ‐ toward Streamlining Drug Discovery with Patient‐Derived Assays

Advanced Biology, EarlyView.
The review provides a critical up‐to‐date overview of the current landscape of human in vitro models for fatty liver disease, including spheroids, organoids, organ‐on‐a‐chip systems, bioprinted liver constructs and precision‐cut liver slices. Their utility for faithfully modeling different stages of MASLD and MASH are evaluated and their compatibility ...
Sonia Youhanna, Nayere Taebnia, Yingxin Liang, Ningtao Cheng, Yi Wang, Maurice Michel, Volker M. Lauschke   +6 more
wiley   +1 more source

Engineering the Link: From Genome Interaction Maps to Functional Insight

Advanced Biology, EarlyView.
Advances in chromosome conformation capture have revealed the genome's 3D organization, yet its causal impact on gene regulation remains elusive. This review highlights emerging genome‐engineering tools ‐ zinc fingers, TALEs, and CRISPR‐Cas9 ‐ that enable targeted manipulation of chromatin loops to dissect structure–function relationships. It discusses
Frido Petersen, Simon Westermann, Valeriia Smialkovska, Jan Mathony, Angelika Feldmann, Dominik Niopek   +5 more
wiley   +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