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Therapeutic Oligonucleotides: An Outlook on Chemical Strategies to Improve Endosomal Trafficking
Cells, 2023 The potential of oligonucleotide therapeutics is undeniable as more than 15 drugs have been approved to treat various diseases in the liver, central nervous system (CNS), and muscles.
Priyanka Mangla +2 more
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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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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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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
New England Journal of Medicine, 2022 BACKGROUND The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1 (SOD1 ...
Timothy A. Miller +25 more
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RNA Drug Delivery Using Biogenic Nanovehicles for Cancer Therapy
Frontiers in Pharmacology, 2021 RNA-based therapies have been promising method for treating all kinds of diseases, and four siRNA-based drugs and two mRNA-based drugs have been approved and are on the market now. However, none of them is applied for cancer treatment.
Nuannuan Li +4 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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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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Advances in oligonucleotide drug delivery
Nature reviews. Drug discovery, 2020 Oligonucleotides can be used to modulate gene expression via a range of processes including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation, non-coding RNA inhibition, gene activation and programmed gene editing. As such, these
T. Roberts, R. Langer, M. Wood
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Recent Advances in Oligonucleotide Therapeutics in Oncology
International Journal of Molecular Sciences, 2021 Cancer is one of the leading causes of death worldwide. Conventional therapies, including surgery, radiation, and chemotherapy have achieved increased survival rates for many types of cancer over the past decades.
Haoyu Xiong +2 more
semanticscholar +1 more source
Exploration, normalization, and summaries of high density oligonucleotide array probe level data.
Biostatistics, 2003 In this paper we report exploratory analyses of high-density oligonucleotide array data from the Affymetrix GeneChip system with the objective of improving upon currently used measures of gene expression. Our analyses make use of three data sets: a small
R. Irizarry +6 more
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