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Advances in oligonucleotide drug delivery - Nature
WEBAug 11, 2020 · Nature Reviews Drug Discovery - Oligonucleotide-based drugs have the potential to treat or manage a wide range of diseases. However, the widespread application of such therapies has been...
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Oligonucleotide Therapeutics: From Discovery and Development …
WEBJan 22, 2022 · In this review, we focus on oligonucleotides designed to bind to RNA (ribonucleic acid) or DNA (deoxyribonucleic acid) by Watson–Crick base pairing. Oligonucleotides act on gene expression via various mechanisms. They can target pre-mRNA, mRNA, or non-coding RNA to induce degradation, modulate splicing events, or …
- DOI: 10.3390/pharmaceutics14020260
- Published: 2022/02
- Publication: Pharmaceutics. 2022 Feb; 14(2): 260.
A Review on Commercial Oligonucleotide Drug Products
WEBApr 26, 2024 · Abstract. Oligonucleotide drug products commercially approved in the US and the EU are reviewed. A total of 20 products that includes 1 aptamer, 12 antisense oligonucleotides (ASOs), 6 small interfering ribonucleic acids (siRNAs), and 1 mixture of single-stranded and double-stranded polydeoxyribonucleotides have been identified.
- Studies of oligonucleotide drug review
Antisense technology: an overview and prospectus | Nature …
WEBMar 24, 2021 · Nine single-stranded antisense oligonucleotide (ASO) drugs representing four chemical classes, two mechanisms of action and four routes of administration have been approved for commercial use...
Widening the horizons of oligonucleotide drug platforms - Nature
WEB15 November 2023. Widening the horizons of oligonucleotide drug platforms. Companies that have pioneered platforms based on oligonucleotides targeting RNA and their biopharma partners...
Oligonucleotide Therapeutics: From Discovery and Development …
WEBJan 22, 2022 · In this review we provide an overview of therapeutic oligonucleotides. We present in particular the different types of oligonucleotides and their modes of action, the tissues they target and the routes by which they are administered to patients, and the therapeutic areas in which they are used.
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Advances in oligonucleotide drug delivery - PubMed
WEBPMID: 32782413. PMCID: PMC7419031. DOI: 10.1038/s41573-020-0075-7. Abstract. 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.
Clinical Pharmacology Studies Supporting Oligonucleotide …
WEBJan 27, 2021 · This review assessed studies focused on four topics: (i) drug‐drug interaction (DDI) liability, (ii) organ impairment studies, (iii) immunogenicity, and (iv) cardiac safety. WHAT DOES THIS STUDY ADD TO OUR KNOWLEDGE? In this investigation, the majority of oligonucleotides were for rare diseases.
Recent Advances in Oligonucleotide Therapeutics in Oncology
WEBMar 24, 2021 · Oligonucleotide therapeutics, which include antisense oligonucleotides, small interfering RNAs, and aptamers, show promising clinical outcomes for disease indications such as Duchenne muscular dystrophy, familial amyloid neuropathies, and macular degeneration.
Advances in oligonucleotide drug delivery - PMC - National …
WEBAug 11, 2020 · This Review will provide an overview of oligonucleotide-based drug platforms and focus on recent advances in improving oligonucleotide drug delivery.
Modern approaches to therapeutic oligonucleotide manufacturing
WEBEditor’s summary. Oligonucleotide therapeutics are emerging as a powerful platform for modulating gene expression across a range of diseases and applications. However, their complex chemical synthesis, concerns about stability and purity, and cost have limited their development and use.
WEBREVIEW. THERAPEUTIC OLIGOS. Modern approaches to therapeutic oligonucleotide manufacturing. R. Obexer1 †, M. Nassir2 , E. R. Moody1 , P. S. Baran2 *, S. L. Lovelock1 † † *. Therapeutic oligonucleotides are a powerful drug modality with the potential to treat many diseases.
The current landscape of nucleic acid therapeutics - Nature
WEBMay 31, 2021 · Here, we review four platform technologies that have enabled the clinical translation of nucleic acid therapeutics: antisense oligonucleotides, ligand-modified small interfering RNA conjugates,...
Frontiers | A perspective on oligonucleotide therapy: Approaches …
WEBOct 18, 2022 · The review provides updated literature on the challenges and bottlenecks of OT and recent advancements in OT drug delivery. Further, this review deliberates on a newly emerging approach to personalized treatment for patients with rare and fatal diseases with OT. 1 Introduction.
Delivery of oligonucleotide‐based therapeutics: challenges and ...
WEBReview. 6 April 2021. Delivery of oligonucleotide‐based therapeutics: challenges and opportunities.
Treating Disease at the RNA Level with Oligonucleotides
WEBJan 2, 2019 · Video. Treating Disease at the RNA Level with Oligonucleotides 4m 51s. Diseases as different as spinal muscular atrophy and hyperlipidemias can now be treated with drugs that act on RNA. A new...
Chemistry, structure and function of approved oligonucleotide ...
WEBMar 7, 2023 · Abstract. 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 aptamer against a protein.
Oligonucleotide therapeutics: chemistry, delivery and clinical
WEBAbstract. Oligonucleotide therapeutics have the potential to become a third pillar of drug development after small molecules and protein therapeutics. However, the three approved oligonucleotide drugs over the past 17 years have not proven to be highly successful in a commercial sense.
Current State of Oligonucleotide Therapeutics | Pharmaceutical
WEBLearn about the chemistry, manufacturing, and controls of oligonucleotide drugs, such as siRNA and ASO, which are composed of natural and synthetic nucleotides. Find out the challenges and opportunities for these complex molecules in various diseases and development stages.
RNA-based therapeutics: an overview and prospectus
WEBJul 23, 2022 · Introduction. The antisense oligonucleotides (ASOs) inhibiting protein synthesis in the early 1980s promoted the rapid advances of RNA-based therapeutics [ 1 ]. In the 2000s, the proposal of RNAi...
WEBLearning Objectives. Describe oligonucleotide-based therapeutics and the regulatory challenges. Understand Agency’s thinking in the development of product-specific guidance (PSG) on nusinersen....
Therapeutic RNA-silencing oligonucleotides in metabolic …
WEBFeb 24, 2022 · In this Review, we highlight the various potential applications of oligonucleotide therapeutics in metabolic diseases, focusing on siRNA and ASO (siRNA/ASO) drugs that are approved and marketed...
The Challenges and Strategies of Antisense Oligonucleotide Drug ...
WEBApr 16, 2021 · Abstract. Antisense oligonucleotides (ASOs) are used to selectively inhibit the translation of disease-associated genes via Ribonuclease H (RNaseH)-mediated cleavage or steric hindrance. They are being developed as a novel and promising class of drugs targeting a wide range of diseases.
Mitigating Risks in Oligonucleotide Therapeutic Development
WEB3 days ago · Our market research has identified 430 active oligonucleotide research programs spanning six therapeutic areas, with 448 drugs in development focusing on three major modalities: antisense ...
Potential of Cell-Penetrating Peptide-Conjugated Antisense
WEBMay 6, 2024 · Spinal muscular atrophy (SMA) is a severe neuromuscular disorder that is caused by mutations in the survival motor neuron 1 (SMN1) gene, hindering the production of functional survival motor neuron (SMN) proteins. Antisense oligonucleotides (ASOs), a versatile DNA-like drug, are adept at binding to target RNA to prevent translation or …
[Oligonucleotide drugs and their progress in stomatology]
WEBJun 9, 2023 · Oligonucleotide drugs have the characteristics of targeting, modifiability and high biosafety. Recent studies have shown that oligonucleotide can be used to make biosensors, vaccine adjuvants, and has the functions of inhibiting alveolar bone resorption, promoting jaw and alveolar bone regeneration, anti-tumor, destroying plaque biofilm, …
Solid Lipid Nanoparticles for Pulmonary Delivery of …
WEB1 day ago · Biopharmaceuticals such as nucleic acids, proteins, and peptides constitute a new array of treatment modalities for chronic ailments. Invasive routes remain the mainstay of administering biopharmaceuticals due to their labile nature in the biological environment. However, it is not preferred for long-term therapy due to the lack of patient adherence …
A perspective on oligonucleotide therapy: Approaches to patient ...
WEBOct 19, 2022 · The review provides updated literature on the challenges and bottlenecks of OT and recent advancements in OT drug delivery. Further, this review deliberates on a newly emerging approach to personalized treatment for …
Fabrication of Metal Nuclear Acid Framework to Enable …
WEB1 day ago · These high-temperature synthesis conditions limit MNFs' use in delivering large molecular drugs, confining them to small molecule drugs and CpG oligonucleotides. [ 31 , 32 ] Moreover, MNFs produced using high-temperature methods lack targeting specificity and stability, degrading rapidly under physiological conditions, and impairing MNAzyme ...
Effect of genomic and cellular environments on gene expression …
WEBMay 24, 2024 · Gene expression is noisy, even among individual cells from an isogenic population [].Noisy gene expression leads to variable cellular outcomes in differentiation [2,3,4,5], the response to environmental stimuli [6, 7], viral latency [], and chemotherapeutic drug resistance [9,10,11].Explaining the causes of noisy expression remains an …
Oligonucleotide probes for imaging and diagnosis of bacterial ...
WEBTraditional molecular probes are typically created to image infected tissue without accurately identifying the pathogen. In contrast, oligonucleotides can be tailored to target specific RNA sequences, allowing for the identification of pathogens, and even generating antibiotic susceptibility profiles by focusing on drug resistance genes ...
Foundation for Angelman Syndrome Therapeutics | FAST
WEB22 hours ago · The company is creating a drug development ecosystem to bring transformative therapeutics to those living with Angelman syndrome (AS). ... to advance the first investigational antisense oligonucleotide (ASO) through early stage research, IND-enabling studies, and eventually into a Phase 1/2 clinical trial. ... 2023 Year in Review …
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