Results 1 to 10 of about 102,021 (242)

Antisense oligonucleotides for neurodegeneration [PDF]

open access: yesScience, 2020
Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
openaire   +4 more sources

Antisense oligonucleotides [PDF]

open access: yesNeurology Genetics, 2019
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
openaire   +3 more sources

Antisense oligonucleotides in neurological disorders [PDF]

open access: yesTherapeutic Advances in Neurological Disorders, 2018
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
openaire   +3 more sources

Regulation of metabolism by miR-378 [PDF]

open access: yes, 2014
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.
core   +1 more source

Effect of Terminal Groups of Dendrimers in the Complexation with Antisense Oligonucleotides and Cell Uptake [PDF]

open access: yes, 2016
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
core   +1 more source

The role of the RACK1 ortholog Cpc2p in modulating pheromone-induced cell cycle arrest in fission yeast [PDF]

open access: yes, 2013
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
core   +14 more sources

Locked nucleic acid: tighter is different [PDF]

open access: yes, 2013
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
core   +1 more source

Synthesis and anti-HIV activity of thiocholesteryl-coupled phosphodiester antisense oligonucleotides incorporated into immunoliposomes [PDF]

open access: yes, 1994
Encapsulation of oligonucleotides in antibody-targeted liposomes (immunoliposomes) which bind to target cells permits intracellular delivery of the oligonucleotides.
Akhtar   +42 more
core   +1 more source

Mechanisms of the inhibition of reverse transcription by unmodified and modified antisense oligonucleotides [PDF]

open access: yes, 1993
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
core   +1 more source

Micro-RNAS that control myosin expression and myofiber identity [PDF]

open access: yes, 2015
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
core   +1 more source

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