Results 111 to 120 of about 270,275 (243)
Highly loaded nanoparticulate carrier using an hydrophobic antisense oligonucleotide complex
, 1999 Antisense oligonucleotides, and particularly those with phosphorothioate backbones, have emerged as potential gene specific therapeutic agents and are currently undergoing evaluation in clinical trials for a variety of diseases.
Stein, Cy A. +3 more
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Nature CommunicationsDominant negative pathogenic variants in KIF1A result in an allelically heterogeneous neurodegenerative condition that manifests as a variable clinical phenotype including seizures, cognitive deficits, optic nerve atrophy, spasticity, and peripheral ...
Michael V. Zuccaro +13 more
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WO 2014203008 A1 Antisense oligonucleotide compositions
, 2014 The present invention relates to antisense oligonucleotide (ASO) compositions and particularly to compositions and methods for the cytosolic delivery of antisense oligonucleotides (ASOs).
Mitchell, John +2 more
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, 1995 : Corticotropin‐releasing factor (CRF) appears to integrate the endocrine, autonomic, immunologic, and behavioral responses of mammals to stress. To investigate further the role of CRF in the CNS, we have begun investigating the usefulness of “antisense ...
Owens, Michael J +9 more
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Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis
Drug Design, Development and Therapy, 2019 Veena Mathew,1 Annabel K Wang1,2 1Department of Neurology, UCI ALS and Neuromuscular Center, University of California, Irvine, Orange, CA, USA; 2Neurology Section, Tibor Rubin VA Medical Center, Long Beach, CA, USA Abstract: Hereditary transthyretin ...
Mathew V, Wang AK
doaj
Antisense oligonucleotide development for the treatment of muscular dystrophies
, 2015 Introduction: Depending upon the chemistry and annealing target, antisense oligonucleotides can be used to modify gene expression through several distinct modes of action.
Bao, T.L. +3 more
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Bioconjugation strategies for oligonucleotide functionalisation
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
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Evaluating the Specificity of Antisense Oligonucleotide Conjugates: A DNA ARRAY ANALYSIS
, 2002 Antisense oligonucleotides are potentially powerful tools for selective control of cellular and viral gene expression. Crucial to successful application of this approach is the specificity of the oligonucleotide for the chosen RNA target.
Fisher, Michael H. +5 more
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Imaging-Assisted Antisense Oligonucleotide Delivery for Tumor-Targeted Gene Therapy
Antisense oligonucleotide (ASO) represents a class of practical tools for targeting undruggable oncogenes with several candidates currently undergoing clinical investigation. The advancement of antisense therapeutics necessitates comprehensive approaches
Hanwen Liao (18396321) +6 more
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, 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
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