Results 91 to 100 of about 584,539 (379)

Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Spinocerebellar ataxia type 3 (SCA3) is a genetically defined ataxia. The Scale for Assessment and Rating of Ataxia (SARA) is a clinician‐reported outcome that measures ataxia severity at a single time point. In its standard application, SARA fails to capture short‐term fluctuations, limiting its sensitivity in trials.
Marcus Grobe‐Einsler, Vivian Maas, Arian Taheri Amin, Jennifer Faber, Tamara Schaprian, Katharina Hill, Matthis Synofzik, Lisa H. Graf, Heike Jacobi, Manuela Lima, Ana F. Ferreira, Bart P. van de Warrenburg, Ilse H. J. Willemse, Dagmar Timmann, Andreas Thieme, Paola Giunti, Hector Garcia‐Moreno, Magda M. Santana, Jeannette Hübener‐Schmid, Elin H. Davies, Thomas Klockgether   +20 more
wiley   +1 more source

Generation of Choline for Acetylcholine Synthesis by Phospholipase D Isoforms [PDF]

, 2001
DEDICATION: This article is dedicated to the memory of Sue Kim Hanson, a graduate student in the department of Pathology and Laboratory Medicine at Boston University School of Medicine, who perished in the terrorist attacks of September 11, 2001 ...
Blusztajn, Jan Krzysztof, Frohman, Michael A, Zhao, Di   +2 more
core   +3 more sources

Functional and Structural Evidence of Neurofluid Circuit Aberrations in Huntington Disease

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Disrupted neurofluid regulation may contribute to neurodegeneration in Huntington disease (HD). Because neurofluid pathways influence waste clearance, inflammation, and the distribution of central nervous system (CNS)–delivered therapeutics, understanding their dysfunction is increasingly important as targeted treatments emerge.
Kilian Hett, Abigail Dubois, Melanie Leguizamon, Alexander Song, Paula Trujillo, Colin D. McKnight, Ciaran M. Considine, Manus J. Donahue, Daniel O. Claassen   +8 more
wiley   +1 more source

Enhanced antigen presentation and immunostimulation of dendritic cells using acid-degradable cationic nanoparticles. [PDF]

, 2005
Acid-degradable cationic nanoparticles encapsulating a model antigen (i.e., ovalbumin) were prepared by inverse microemulsion polymerization with acid-cleavable acetal cross-linkers.
Fréchet, Jean MJ, Goh, Sarah L, Kwon, Young Jik, Standley, Stephany M   +3 more
core  

Carbon nanotube decorated magnetic microspheres as an affinity matrix for biomolecules [PDF]

, 2013
Carbon nanotube (CNT) decorated magnetic microspheres were fabricated to develop a multimodal platform that utilizes non-covalent molecular interactions of CNTs to magnetically separate biomolecules.
Kolkar Mohammed, Javed Hussain Niazi, Ünal, Hayriye   +1 more
core   +1 more source

Intracellular Trafficking and Endosomal Release of Oligonucleotides: What We Know and What We Don’t

Nucleic Acid Therapeutics, 2018
Understanding the cellular uptake and intracellular trafficking of oligonucleotides provides an important basic underpinning for the developing field of oligonucleotide-based therapeutics.
Rudy L. Juliano
semanticscholar   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Nucleic Acid Metabolism in Patients with Severe Burn Injury and the Possibility of Its Correction

Общая реаниматология, 2019
Purpose. To assess changes in nucleic acid (NA) metabolism in severe burn injury and to develop recommendations for correction of the alimentary status taking into account changes in the NA metabolism. Materials and methods.
E. I. Vereshchagin, I. M. Mitrofanov, I. Yu. Samatov, E. I. Strel'tsova, A. L. Veinberg, E. A. Potter, S. S. Bogachov   +6 more
doaj   +1 more source

Managing the sequence-specificity of antisense oligonucleotides in drug discovery

Nucleic Acids Research, 2017
All drugs perturb the expression of many genes in the cells that are exposed to them. These gene expression changes can be divided into effects resulting from engaging the intended target and effects resulting from engaging unintended targets.
P. Hagedorn, B. Hansen, T. Koch, M. Lindow   +3 more
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