Chemistry, structure and function of approved oligonucleotide therapeutics
Nucleic Acids Research, 2023 Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi) and an RNA ...
M. Egli, M. Manoharan
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Targeted tissue delivery of RNA therapeutics using antibody–oligonucleotide conjugates (AOCs)
Nucleic Acids Research, 2023 Although targeting TfR1 to deliver oligonucleotides to skeletal muscle has been demonstrated in rodents, effectiveness and pharmacokinetic/pharmacodynamic (PKPD) properties remained unknown in higher species.
Barbora Malecova +14 more
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Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
EMBO Molecular Medicine, 2021 Nucleic acid‐based therapeutics that regulate gene expression have been developed towards clinical use at a steady pace for several decades, but in recent years the field has been accelerating.
S. Hammond +21 more
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The Application of Microfluidic Technologies in Aptamer Selection
Frontiers in Cell and Developmental Biology, 2021 Aptamers are sequences of single-strand oligonucleotides (DNA or RNA) with potential binding capability to specific target molecules, which are increasingly used as agents for analysis, diagnosis, and medical treatment.
Yang Liu +14 more
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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.
Scoles, Daniel R. +2 more
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Development of a sensitive ELISA to quantify apolipoprotein CIII in nonhuman primate serum
Journal of Lipid Research, 2011 Apolipoprotein CIII (apoCIII), a major constituent of triglyceride-rich lipoprotein, has been proposed as a key contributor to hypertriglyceridemia on the basis of its inhibitory effects on lipoprotein lipase.
Yuxin Wang +4 more
doaj +1 more source
Microarray-based ultra-high resolution discovery of genomic deletion mutations [PDF]
, 2014 BACKGROUND: Oligonucleotide microarray-based comparative genomic hybridization (CGH) offers an attractive possible route for the rapid and cost-effective genome-wide discovery of deletion mutations.
Baban, D. +8 more
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Oligonucleotide Therapeutics: From Discovery and Development to Patentability
Pharmaceutics, 2022 Following the first proof of concept of using small nucleic acids to modulate gene expression, a long period of maturation led, at the end of the last century, to the first marketing authorization of an oligonucleotide-based therapy.
L. Moumné +2 more
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Nucleic acid amphiphiles: Synthesis, properties, and applications
Molecular Therapy: Nucleic Acids, 2023 Nucleic acid amphiphiles, referring to nucleic acids modified with large hydrophobic groups, have been widely used in programmable bioengineering. Since nucleic acids are intrinsically hydrophilic, the hydrophobic groups endow nucleic acid amphiphiles ...
Amu Gubu +5 more
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Investigating synthetic oligonucleotide targeting of miR31 in Duchenne muscular dystrophy [PDF]
, 2016 Exon-skipping via synthetic antisense oligonucleotides represents one of the most promising potential therapies for Duchenne muscular dystrophy (DMD), yet this approach is highly sequence-specific and thus each oligonucleotide is of benefit to only a ...
Hildyard, J C W, Wells, D J
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