Results 81 to 90 of about 430,879 (296)
Nucleic Acid Metabolism in Patients with Severe Burn Injury and the Possibility of Its Correction
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 +6 more
doaj +1 more source
Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points
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 +20 more
wiley +1 more source
Relevance. Studies on model systems have confirmed the effectiveness of antisense oligonucleotides, including those that contain photoactive groups, for the modification of nucleic acids.
Asya S. Levina +5 more
doaj +1 more source
We report the structure activity relationships of short 14-mer phosphorothioate gapmer antisense oligonucleotides (ASOs) modified with α-L-locked nucleic acid (LNA) and related modifications targeting phosphatase and tensin homologue (PTEN) messenger RNA
Punit P Seth +5 more
doaj +1 more source
Functional and Structural Evidence of Neurofluid Circuit Aberrations in Huntington Disease
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 +8 more
wiley +1 more source
Low allelic and clonal variability among endogenous RNAi targets has focused mismatch tolerance studies to RNAi-active guide strands. However, the inherent genomic instability of RNA viruses such as hepatitis C virus (HCV) gives rise to quasi-species ...
Pantazis I. Theotokis +4 more
doaj +1 more source
Functional analysis of human alpha 1(I) procollagen gene promoter. Differential activity in collagen-producing and -nonproducing cells and response to transforming growth factor beta 1. [PDF]
To gain a further understanding of the regulation of human type I collagen gene expression under physiologic and pathologic conditions, we characterized 5.3 kilobase pairs (kb) of the human alpha 1(I) procollagen gene promoter.
Diaz, Arturo +6 more
core +1 more source
Oligonucleotides are (short) chains of (chemically modified) ribo- or deoxyribonucleotides. Their ability to bind to chromosomal DNA, mRNA, or non-coding RNA (ncRNA) through Watson-Crick and Hoogsteen base pairing offers possibilities for highly specific intervention in gene transcription, mRNA translation, gene repair, and recombination for ...
Schiffelers, R.M., Mastrobattista, E.
openaire +4 more sources
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 +12 more
wiley +1 more source
A sequence‐encoded supramolecular construct containing two accessible toeholds is developed herein for enabling multiple editing operations. By introducing specific input strands, it is possible to selectively erase or rewrite digital content through parallel or series toehold‐mediated strand displacement (PTMSD or STMSD).
Jakub Ossowski +3 more
wiley +1 more source

