Results 111 to 120 of about 171,175 (241)
Gas-phase fragmentation and conformation of the sugar-modified antisense oligonucleotide tcDNA
, 2015 Tricyclo-DNA (tcDNA) is a sugar- and backbone-modified analogue of DNA that is currently tested as antisense oligonucleotide for the treatment of Duchenne muscular dystrophy.
Hari, Yvonne, Schürch, Stefan
core
Blood SOD1 Activity in ALS Patients Receiving Tofersen Treatment
Annals of Neurology, EarlyView.Objective The antisense oligonucleotide tofersen is the first disease‐modifying drug for SOD1‐related amyotrophic lateral sclerosis (ALS) and was approved because of its ability to reduce SOD1 protein and neurofilament levels. The effect of tofersen on SOD1 activity is unclear but of clinical relevance because homozygous SOD1 mutations, linked to ...
Katharina Goehring +18 more
wiley +1 more source
Annals of Neurology, EarlyView.Objective To characterize magnetic resonance imaging (MRI)‐based glymphatic surrogates in Huntington's disease (HD) using MRI measures of perivascular diffusivity and structural perivascular alterations across multiple large cohorts. Methods We analyzed 2,731 MRI sessions from 880 participants across 3 large retrospective HD cohorts.
Alexia Solomon +5 more
wiley +1 more source
Bioconjugation strategies for oligonucleotide functionalisation [PDF]
Chemical modification of oligonucleotides has gained growing attention in research and therapeutic areas as these modalities, such as ribose modifications or fluorescent tags, have shown great promise to improve therapeutic properties and enable real ...
Chik, Gerald
core +1 more source
Analytical HPLC analysis of purified antisense oligonucleotide.
, 2013 Sample preparation: 25 µL purified antisense oligonucleotide; Column: DNAPac PA-100 (4/250); Flow rate: 1 ml/min; Buffer A: 10 mM NaClO4+1 mM Tris; Buffer B: 300 mM NaClO4+1 mM Tris; Gradient: 10–70% B, 7.6 CV.
Shuang-yong Xu (14559) +2 more
core +1 more source
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 +4 more
wiley +1 more source
Inhibition of pteridine reductase 1 (PTR1) expression in Leishmania promastigotes using a full-length antisense construct [PDF]
, 2009 Leishmania exhibit many unusual features, one of which is the pteridine metabolic pathway essential for the growth for these parasites. Leishmania have evolved a complex and versatile pteridine salvage network capable of scavenging a wide array of ...
Nariman Mosaffa +7 more
core
British Journal of Clinical Pharmacology, EarlyView.Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić +4 more
wiley +1 more source
Oligonucleotide N3'-->P5' phosphoramidates as antisense agents. [PDF]
, 1996 Uniformly modified oligonucleotide N3'-->P5' phosphoramidates, where every 3'-oxygen is replaced by a 3'-amino group, were synthesized. These compounds have very high affinity to single-stranded RNAs and thus have potential utility as antisense agents.
CALABRETTA, Bruno +8 more
core
Precision medicine in paediatrics: Progress and priorities
British Journal of Clinical Pharmacology, EarlyView.Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source