Antisense oligonucleotides for neurodegeneration [PDF]
Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
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Antisense oligonucleotides [PDF]
There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine.
Eric Vallabh Minikel+2 more
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Antisense oligonucleotides in neurological disorders [PDF]
The introduction of genetics revolutionized the field of neurodegenerative and neuromuscular diseases and has provided considerable insight into the underlying pathomechanisms. Nevertheless, effective treatment options have been limited. This changed recently when antisense oligonucleotides (ASOs) could be translated from in vitro and experimental ...
Albert C. Ludolph, Claudia D. Wurster
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Regulation of metabolism by miR-378 [PDF]
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.
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Effect of Terminal Groups of Dendrimers in the Complexation with Antisense Oligonucleotides and Cell Uptake [PDF]
Indexación: Web of Science.Poly(amidoamine) dendrimers are the most recognized class of dendrimer. Amino-terminated (PAMAM-NH2) and hydroxyl-terminated (PAMAM-OH) dendrimers of generation 4 are widely used, since they are commercially available.
Araya-Durán, Ingrid+10 more
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The role of the RACK1 ortholog Cpc2p in modulating pheromone-induced cell cycle arrest in fission yeast [PDF]
The detection and amplification of extracellular signals requires the involvement of multiple protein components. In mammalian cells the receptor of activated C kinase (RACK1) is an important scaffolding protein for signal transduction networks. Further,
A Goddard+61 more
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Locked nucleic acid: tighter is different [PDF]
This viewpoint briefly reviews the impact of Locked Nucleic Acid (LNA) oligonucleotides, first described in a ChemComm paper in 1998. A number of unique applications in oligonucleotide biotechnology have been made possible by the high binding affinity ...
Watts, Jonathan
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Synthesis and anti-HIV activity of thiocholesteryl-coupled phosphodiester antisense oligonucleotides incorporated into immunoliposomes [PDF]
Encapsulation of oligonucleotides in antibody-targeted liposomes (immunoliposomes) which bind to target cells permits intracellular delivery of the oligonucleotides.
Akhtar+42 more
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Mechanisms of the inhibition of reverse transcription by unmodified and modified antisense oligonucleotides [PDF]
We demonstrated that unmodified and modified (phosphorothioate) oligonucleotides prevent cDNA synthesis by AMV or HIV reverse transcriptases. Antisense oligonucleotide/RNA hybrids specifically arrest primer extension.
Hatta, Toshifumi+6 more
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Micro-RNAS that control myosin expression and myofiber identity [PDF]
The present invention relates to the identification of two microRNAs, miR-499 and miR-208b, that repress fast skeletal muscle contractile protein genes. Expression of miR-499 and/or miR-208b can be used to repress fast fiber genes and activate slow fiber
Olson, Eric N., van Rooij, Eva
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