Results 11 to 20 of about 124,170 (327)
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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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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Antisense oligonucleotides as therapeutic agents [PDF]
Journal of Cellular Physiology, 1999 Antisense oligonucleotides can block the expression of specific target genes involved in the development of human diseases. Therapeutic applications of antisense techniques are currently under investigation in many different fields. The use of antisense molecules to modify gene expression is variable in its efficacy and reliability, raising objections ...
Umberto Galderisi +2 more
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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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Beilstein Journal of Organic Chemistry, 2021 Chemical modifications have been extensively used for therapeutic oligonucleotides because they strongly enhance the stability against nucleases, binding affinity to the targets, and efficacy. We previously reported that oligonucleotides modified with an
Naohiro Horie +3 more
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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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Expression of ssDNA in Mammalian Cells
BioTechniques, 2003 Antisense therapy involves the use of antisense oligonucleotides for altering targeted gene function. However, the low efficiency of cell delivery of antisense oligonucleotides has limited the efficacy of antisense therapeutic approaches.
Yin Chen, Yong-Jie Ji, Charles Conrad
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Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease
Molecules, 2017 Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting.
Valentina Sardone +4 more
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Polymerase-endonuclease amplification reaction (PEAR) for large-scale enzymatic production of antisense oligonucleotides. [PDF]
PLoS ONE, 2010 Antisense oligonucleotides targeting microRNAs or their mRNA targets prove to be powerful tools for molecular biology research and may eventually emerge as new therapeutic agents.
Xiaolong Wang +2 more
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