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Antisense oligonucleotides in neurological disorders [PDF]
Therapeutic 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.
Claudia D. Wurster, Albert C. Ludolph
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Antisense oligonucleotides for neurodegeneration [PDF]
Science, 2020 Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
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Antisense oligonucleotides [PDF]
Neurology 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
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Accessible light-controlled knockdown of cell-free protein synthesis using phosphorothioate-caged antisense oligonucleotides [PDF]
Communications Chemistry, 2023 Light-activated antisense oligonucleotides have been developed to induce gene knockdown in living cells, however, their synthesis remains challenging and application in cell-free systems is underexplored.
Denis Hartmann, Michael J. Booth
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Molecular Mechanisms of Antisense Oligonucleotides [PDF]
Nucleic Acid Therapeutics, 2017 In 1987, when I became interested in the notion of antisense technology, I returned to my roots in RNA biochemistry and began work to understand how oligonucleotides behave in biological systems. Since 1989, my research has focused primarily on this topic, although I have been involved in most areas of research in antisense technology.
S. Crooke
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Antisense oligonucleotide inhibition of Heat Shock Protein (HSP) 47 improves bleomycin-induced pulmonary fibrosis in rats [PDF]
Respiratory Research, 2007 Background The most common pathologic form of pulmonary fibrosis arises from excessive deposition of extracellular matrix proteins such as collagen. The 47 kDa heat shock protein 47 (HSP47) is a collagen-specific molecular chaperone that has been shown ...
Noguchi Takayuki +3 more
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Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models [PDF]
Journal of Clinical Investigation, 2018 Mutations in superoxide dismutase 1 (SOD1) are responsible for 20% of familial ALS. Given the gain of toxic function in this dominantly inherited disease, lowering SOD1 mRNA and protein is predicted to provide therapeutic benefit.
A. McCampbell +22 more
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Tissue pharmacokinetics of antisense oligonucleotides
Molecular Therapy: Nucleic AcidsPharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue.
Erica Bäckström +7 more
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Biomedicines, 2023 Cancer is one of the leading causes of death globally. Epidermal growth factor receptor is one of the proteins involved in cancer cell proliferation, differentiation, and invasion. Antisense oligonucleotides are chemical nucleic acids that bind to target
Akilandeswari Ashwini Balachandran +3 more
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Nucleic Acids Research, 2021 The PS modification enhances the nuclease stability and protein binding properties of gapmer antisense oligonucleotides (ASOs) and is one of very few modifications that support RNaseH1 activity.
Brooke A. Anderson +16 more
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