Therapeutic oligonucleotides. Medical search. Frequent questions

The U.S. Pharmacopoeia (USP) Therapeutic Peptides and Oligonucleotides: Regulations, Standards and Quality workshop is a 2-day event taking place November 4-5 in Rockville, Maryland. (epivax.com)
This workshop continues USP's mission to inform public standard setting for therapeutic peptides and peptide vaccines while introducing similar considerations for therapeutic oligonucleotides. (epivax.com)
Aptamers are oligonucleotides or peptides that bind to a specific target molecule. (labwrench.com)

Our innovative therapeutic platform for blood-to-brain delivery of ASOs may revolutionize management of neurodegenerative diseases,' senior author Takanori Yokota claims. (news-medical.net)
Another growing area of research in the Andaloussi lab pertains to the delivery of antisense oligonucleotides (ASOs) that are used to either silence genes or induce splice switching. (varbi.com)
A major focus is on enhancing delivery of therapeutic ASOs to heart and CNS to treat genetic diseases. (varbi.com)
This project, which is funded by a recently awarded Novo Nordisk Distinguished Innovator grant, seek to unlock functional cardiac delivery of therapeutic ASOs. (varbi.com)
Therapeutic tiny or short modified antisense oligonucleotides (Tiny ASOs) can block gene expression or modulate splice-switching! (biosyn.com)
Antisense oligonucleotides (ASOs) have proven to be an effective therapeutic platform for the treatment of disease. (canal-u.tv)
Antisense oligonucleotides (ASOs) composed of nucleic acid mimics (NAMs) monomers are considered as potential novel therapeutic drugs against bacterial infections. (bvsalud.org)

30 August 2023 -- Cambridge, UK -- Intellegens, a company applying machine learning (ML) to accelerate innovation in R&D, announced that it has secured a £1.6m grant from Innovate UK, part of UK Research and Innovation (UKRI), to apply ML in the emerging field of oligonucleotide therapies. (iptonline.com)

An approach to therapeutic modulation utilizes agents that modulate endogenous transcription by specifically targeting those genes at the gDNA level. (wikipedia.org)
Oligonucleotide-based drugs have received considerable attention for their capacity to modulate gene expression very specifically and as a consequence they have found applications in the treatment of many human acquired or genetic diseases. (ox.ac.uk)
In addition to ultrasound contrast, perfluorocarbon-exposed sonicated dextrose albumin (PESDA) microbubbles (MB) are capable of binding agents which modulate gene expression like antisense oligonucleotides (AON). (nebraska.edu)
Oligonucleotides modulate gene-specific expression within cells and can be used to identify genes involved in diseases. (waterstones.com)

Therapeutic oligonucleotides are synthetic oligonucleotides composed of 10-30 nucleic acid bases. (shimadzu.com)
Modulation of gene expression can be mediated at the level of transcription by DNA-binding agents (which may be artificial transcription factors), small molecules, or synthetic oligonucleotides. (wikipedia.org)
There are three major categories of agents that act as transcriptional gene modulators: triplex-forming oligonucleotides (TFOs), synthetic polyamides (SPAs), and DNA binding proteins. (wikipedia.org)
The Medicines Manufacturing Innovation Centre launched a collaboration with AstraZeneca, Exactmer, Novartis, and UK Research and Innovation to develop a scalable, sustainable, and more cost-effective medicines manufacturing process for oligonucleotides: short strands of synthetic DNA or RNA. (mordorintelligence.com)
As a consequence of their highly serial synthesis and use of modified nucleotides, oligonucleotides are uniquely susceptible to fairly high levels of synthetic impurities. (biopharminternational.com)
87(5): 1663-1667] Specifically, the investigators started by priming whole cerebellar RNA with a synthetic oligonucleotide containing a T7 RNA polymerase promoter sequence. (genomicglossaries.com)
Synthetic antisense RNAs are used to effect the functioning of specific genes for investigative or therapeutic purposes. (genomicglossaries.com)
Therapeutic oligonucleotides are large, complex molecules, requiring a multi-stage synthetic process alongside significant purification and analysis steps. (iptonline.com)

For instance, in September 2021, SpeeDx invested in local oligonucleotide manufacturing to reduce supply chain risk and develop a more vital sovereign capacity for critical diagnostics. (mordorintelligence.com)
For instance, in February 2021, CPI launched a project in the Medicines Manufacturing Innovation Centre to revolutionize the manufacture of oligonucleotides. (mordorintelligence.com)

Oligonucleotide therapeutics are mainly based on artificial oligonucleotides that are chemically modified DNA or RNA, which improves their structural stability in the human body and affinity to their target molecules 1 ). (jascoinc.com)

The oligonucleotide synthesis market is expected to register a CAGR of 12.3% over the forecast period. (mordorintelligence.com)
COVID-19 has impacted the growth of the oligonucleotide synthesis market. (mordorintelligence.com)
Oligonucleotide Synthesis Market was valued at US$ 5.75 Bn in 2022 and is expected to reach 15.20Bn by 2029, at a CAGR of 14.87% during the forecast period. (maximizemarketresearch.com)
The oligonucleotide synthesis market has found major development in the recent years and is anticipated to grow tremendously during the forecast period. (maximizemarketresearch.com)
The oligonucleotide synthesis market is expected to witness limitations in the availability of funding for research in academic laboratories, which may hamper market revenue growth. (maximizemarketresearch.com)
The report also helps in understanding Global Oligonucleotide Synthesis Market dynamics, structure by identifying and analyzing the market segments and project the global market size. (maximizemarketresearch.com)
1. Global Oligonucleotide Synthesis Market analysis and forecast, in terms of value. (maximizemarketresearch.com)

Given that phosphorothioate modifica-tion is the most common method of enhancing oligonucleotide stability in vivo, we expect MRM-based assays constructed by a similar methodology to be applicable across a variety of therapeutic oligonucleotides. (shimadzu.com)

ERG), Immunostaining monitors therapeutic effect, immunoassays track biomarkers, and qRT-PCR provides information on retinal gene expression. (pharmoptima.com)

More specifically, antisense oligonucleotides that are useful as reagents for target validation , or as drugs, are engineered molecules that differ from natural RNA but that have a base sequence that is recognized as being complementary to a very specific mRNA sequence. (genomicglossaries.com)
Oligonucleotides are complex molecules and the process of bringing them to market is costly, meaning very few companies are able to harness this potentially life-saving and life-changing technology. (iptonline.com)

The symposium will address therapeutic, diagnostic, analytical as well as basic research applications of aptamers and invites proposals for podium (now closed! (libpubmedia.co.uk)

In this review, we focus on early stages of the disease and we address both biomarkers and therapeutic approaches currently available and under research. (hindawi.com)

Other significant factors include the increasing government investments and R&D expenditure in pharmaceutical and biotechnology companies and the use of synthesized oligonucleotides in molecular diagnostics and clinical applications. (mordorintelligence.com)
Additionally, the rising company and government initiatives to boost the development of oligonucleotide-based drug products and the adoption of oligonucleotide technology in the pharmaceutical and biotechnology industry are also expected to increase market growth. (mordorintelligence.com)
Pharmaceutical biology antisense, antisense DNA, antisense oligonucleotides. (genomicglossaries.com)
The biotechnology & Pharmaceutical Companies segment is expected to lead the market for oligonucleotide synthesis. (maximizemarketresearch.com)
Offering high specificity with the ability to treat an extremely broad range of diseases, oligonucleotides represent a promising therapeutic option in pharmaceutical research. (iptonline.com)
Through CPI's Medicines Manufacturing Innovation Centre, we will look to bring together a community of experts from the pharmaceutical and technology companies to support the development of the Alchemite tools, reducing development times and improving manufacturability of oligonucleotide therapeutics. (iptonline.com)

The MBO also potentiates the therapeutic effects of chemotherapeutic agents and radiation therapy in various tumors, through both p53-dependent and p53-independent mechanisms, indicating that MDM2 inhibitors have a broad spectrum of anti-tumor activity in human cancers, regardless of p53 status. (nih.gov)
[6] Their targeting ability was believed to limit side effects for cancer patients and give a wider therapeutic window than other chemotherapeutic agents, although this promise hasn't yet been realized in the clinic. (wikipedia.org)

Considering the complexity of MDM2 functions, we have chosen to use gene silencing technologies including antisense oligonucleotides and RNA interference. (nih.gov)
Antisense mixed-backbone oligonucleotides (MBO) specifically inhibit MDM2 expression in a dose- and time-dependent manner, resulting in significant anti-tumor activity in vitro and in vivo. (nih.gov)
These results provide a basis for clinical evaluation of antisense anti-MDM2 oligonucleotides as chemosensitizers and radiosensitizers. (nih.gov)

Antisense oligonucleotide (ASO) therapy has the potential to ameliorate many neurodegenerative diseases at the genetic level to suppress the production of harmful proteins or non-coding RNAs. (news-medical.net)
Background Therapeutic targeting of RNA may be a strategy which could inhibit the translation of one or more disease-associated proteins. (sbir.gov)

We may also strategize by initiating treatment with intrathecal dosing to rapidly achieve therapeutic concentrations, followed by intravenous or subcutaneous maintenance as needed. (news-medical.net)
Triplex-forming oligonucleotides (TFO) are one potential method to achieve therapeutic gene modulation. (wikipedia.org)

The purpose of this study is to examine the therapeutic efficacy of human umbilical cord mesenchymal stem/stromal cells (UMSCs) in treating congenital and acquired corneal opacity associated with the loss of collagen V. (molvis.org)

many promising therapeutic approaches are still clinically unavailable. (varbi.com)
Gene therapy involves viral, plasmid, and oligonucleotide-based approaches. (medscape.com)
The relationship between miRNAs and cancer was demonstrated for the first time in 2002, with miRNAs being stated as a potential mechanism that may contribute to improve some cancer therapeutic approaches through restoring or blocking the miRNAs function ( 11 ). (frontiersin.org)
Currently, RT is one of the most often used therapeutic approaches in PCa patients, featured by several levels of complexity ( 13 , 14 ), with around 50% of all PCa patients receiving RT at some stage of treatment, while 10-45% of PCa cases are resistant to irradiation ( 15 , 16 ). (frontiersin.org)

Quantification of oligonucleotides is complicated by the typical use of ion-pairing reagents. (biopharminternational.com)

Aptamers 2016 will be co-hosted with our 2nd symposium on antisense and therapeutic oligonucleotides, Oligo 2016 , on 6th April 2016. (libpubmedia.co.uk)
Aptamers are useful in biotechnological and therapeutic applications as they offer molecular recognition properties that rival that of the commonly used biomolecule, antibodies. (labwrench.com)
In addition to their discriminate recognition, aptamers offer advantages over antibodies as they can be engineered completely in a test tube, are readily produced by chemical synthesis, possess desirable storage properties, and elicit little or no immunogenicity in therapeutic applications. (labwrench.com)

Validating survivin as a cancer therapeutic target. (biomedcentral.com)

Pandey SK, Ghosh S, Maiti P, Haldar C. Therapeutic efficacy and toxicity of tamoxifen loaded PLA nanoparticles for breast cancer. (springer.com)

Scientists are still refining the technology to turn TFOs into a therapeutic product and much of this revolves around their potential applications in antigene therapy. (wikipedia.org)
In this review, we highlight the latest advances in the field of microRNA biology and their potential as emerging therapeutic targets in liver disease. (nih.gov)
To rapidly prototype potential PMO drug candidates, we report a fully automated flow-based oligonucleotide synthesizer. (nature.com)
Continuing developments in oligonucleotide research have begun to unleash their potential as therapeutic agents. (waterstones.com)
demonstrate through studies on induced pluripotent stem cell-derived cortical neurons that miR-33a is a potential therapeutic target for the treatment of SPG4-related hereditary spastic paraplegia. (portlandpress.com)
Ben Pellegrini, CEO, Intellegens, said: "The potential of oligonucleotide therapeutics to treat a vast spectrum of diseases is an exciting prospect that is unfortunately held back by manufacturing capabilities. (iptonline.com)
Dave Tudor, managing director of Pharmaceuticals at CPI, said: "We are really excited to be working with Intellegens on this project to help harness the potential of oligonucleotides to treat a variety of conditions. (iptonline.com)
The GD 3 Ocular Center of Excellence is proud to offer efficacy models in which physiological readouts coupled with cellular and biochemical measurements provide a comprehensive snapshot of your treatment's therapeutic potential. (pharmoptima.com)

Given its reported efficacy in both types of hemophilia without inhibitors, concizumab is evolving as an attractive therapeutic for all patients with hemophilia. (cdc.gov)

We will focus on the use of antisense oligonucleotides, cell cycle analysis, and luciferase reporter genes. (biomedcentral.com)
La determinación de un patrón de genes expresados al nivel de la TRANSCRIPCIÓN GENÉTICA bajo circunstancias específicas o en una célula específica. (bvsalud.org)

Although the recognition of CpG DNA by B cells and plasmacytoid dendritic cells is mediated by TLR 9, these cell types differ in their ability to bind and respond to structurally distinct classes of CpG oligonucleotides. (fda.gov)
Phosphoryl guanidine oligo-2′-O-methylribonucleotides in neutral therapeutic oligonucleotides enable enhanced cell penetration! (biosyn.com)
however, these technologies rely on derivatized oligonucleotide structures which have poor cell permeability and biodistribution which limits their effectiveness as a therapeutic. (sbir.gov)
Antisense morpholino oligonucleotides directed at intronic pseudoexons have been shown to increase propionyl-CoA carboxylase activity to normal levels in fibroblast cell lines derived from patients suffering from propionic acidemia. (medscape.com)

The collaboration will validate Alchemite as a tool to improve productivity in oligonucleotide process development and manufacturing workflows. (iptonline.com)

Therapeutic strategies for the dystrophinopathies can be categorized into 3 groups based on their approach: (1) Supportive pharmacologic therapy, (2) research gene therapy, and (3) research cellular therapy. (medscape.com)

Oligonucleotides are a chemically distinct class from small-molecule or protein therapeutics and present their own unique challenges for characterization and quantification. (biopharminternational.com)
RNA-like oligonucleotides that are complementary to a portion of a target mRNA molecule. (genomicglossaries.com)

This review focuses on CPP-delivery of several classes of oligonucleotides (ONs), namely antisense oligonucleotides, splice switching oligonucleotides (SSOs) and siRNAs. (ox.ac.uk)
Gewirtz AM, Stein CA, Glazer PM. Facilitating oligonucleotide delivery: helping antisense deliver on its promise. (biomedcentral.com)

Such developments are expected to increase the adoption of oligonucleotides in drug development which is anticipated to fuel the market growth over the forecast period. (mordorintelligence.com)
Recent developments in the market for oligonucleotide synthesis: In March 2018, Danaher Corporation publicized that they have acquired Integrated DNA Technologies. (maximizemarketresearch.com)

The first therapeutic oligonucleotides were developed to treat unusual but serious genetic diseases, but we are now seeing a rapid development for treatment of common diseases as well. (gu.se)
Inhibitory oligodeoxynucleotides - therapeutic promise for systemic autoimmune diseases? (enzolifesciences.com)

In collaboration with the Centre for Process Innovation (CPI), the two-year project will augment and validate the company's ML technology, Alchemite, as a tool to enhance productivity and yields of oligonucleotide therapeutic manufacture through performance prediction and optimisation of quality control strategies. (iptonline.com)

This article describes using a liquid chromatograph/mass spectrometer to determine molecular mass and construct a quantitative assay for a therapeutic oligonucleotide and its analogs. (shimadzu.com)
Based on mass spectral deconvolution of multivalent ions, we determined the molecular weight of the oligonucleotides to a mass error of less than 1 ppm. (shimadzu.com)
The increased demand for COVID-19 diagnostics has, in turn, increased the global demand for oligonucleotide raw materials that form the basis of molecular diagnostic products. (mordorintelligence.com)
Furthermore, the increasing adoption of oligonucleotides in molecular diagnostics and clinical applications contributes to market growth. (mordorintelligence.com)

Also, developing therapeutic COVID-19 medicines employing essential mutational targets, such as the 5'-polyU tract, can lessen the production and spread of new SARS-CoV-2. (mordorintelligence.com)
Therefore, with the aim to summarize the current knowledge on the initiation of the atherosclerotic process, in this paper, we review the early markers of atherosclerosis and we address the main therapeutic targets for preventing atheroma formation at its very initial stages focusing on inflammation, oxidative stress, endothelial dysfunction, and the interaction between platelets and endothelium. (hindawi.com)

Oligonucleotides are short sections of nucleic acids that are united utilizing enzyme chemistry and genetic engineering. (maximizemarketresearch.com)

Following successful completion of this proposal, we will seek further resources to continue advancing this therapeutic strategy toward clinical application. (michaeljfox.org)
We propose scientific development and commercialization activities to bring to a clinical application a therapeutic approach for tumors based on DDRNA inhibition by ASO. (europa.eu)

The research team had recently developed DNA/RNA heteroduplex oligonucleotide (HDO) technology capable of highly efficient RNA degradation in vivo. (news-medical.net)

SMOT III aims to provide a platform to discuss and share success stories of outstanding companies and innovative projects in the field of not only oligonucleotides, but also the development of innovative medicines, medical devices and interesting biotechnological projects. (myscience.es)

Such research studies have impacted the demand and adoption for oligonucleotides in drug development, affecting the market growth during a pandemic. (mordorintelligence.com)
This is expected to increase the demand for oligonucleotides for drug development, boosting the market growth. (mordorintelligence.com)
OligoNova Hub is Sweden's new national infrastructure for drug development, especially focused on therapeutic oligonucleotides. (gu.se)
As a result, these companies are making major use of oligonucleotides, as it offers an effective mode of action for the treatment of several indications at comparatively lower development costs. (maximizemarketresearch.com)

In a study published in PLOS One in March 2022, it was observed that the oligonucleotides successfully targetted 5'-polyUs in the coronavirus genome. (mordorintelligence.com)

These are utilized for diagnostics, therapeutic, and in addition industrial and academic. (maximizemarketresearch.com)

The La Fe Health Research Institute of Valencia, in collaboration with the company Arthex Biotech, have organised the III Spanish Meeting on Oligonucleotide Therapeutics, SMOT III, at the Faculty of Pharmacy of the University of Valencia on October 26 and 27. (myscience.es)
The event brings together experts, the cutting edge in research and entrepreneurship, and investment companies to foster collaboration and promote advances in the field of therapeutic oligonucleotides (ONs). (myscience.es)
Our work demonstrates that RGENs are highly active even with imperfectly matched RNA-DNA interfaces in human cells, a finding that might confound their use in research and therapeutic applications. (cdc.gov)
Understanding the scope of RGEN-mediated off-target effects in human and other eukaryotic cells will be critically essential if these nucleases are to be used widely for research and therapeutic applications. (cdc.gov)

Nucleobases that compose DNA, RNA and artificial oligonucleotides almost all have an absorbance in the UV region, and usually with a peak maxima at around 260 nm. (jascoinc.com)

ASO) aims to induce a long-term decrease in expression of this protein kinase to reduce kinase activity as a therapeutic treatment. (michaeljfox.org)
Through this unique and novel approach, we can develop a long-term, effective and stable therapeutic treatment decreasing LRRK2 kinase activity and alleviating LRRK2-associated neuronal dysfunction in PD. (michaeljfox.org)
The strength of such a strategy is highlighted by the emergence of SARS-CoV-2, wherein oligonucleotide-based drugs were among the first treatment types to enter human trials as early as 2 months after the virus's first reports 4 , 5 , 6 . (nature.com)

One of the treatments applied in cancer is radiotherapy (RT), a therapeutic modality that uses ionizing radiation to induce damage in unwanted cells. (frontiersin.org)

We demonstrate the power of our automated technology with the synthesis of milligram quantities of three candidate therapeutic PMO sequences for an unserved class of Duchenne muscular dystrophy (DMD). (nature.com)
The therapeutic strategies are usually applied first to Duchenne muscular dystrophy with the thought that benefits can be extrapolated to Becker muscular dystrophy. (medscape.com)

This neatly 10-fold increase in antisense inhibition of CYP3A2 expression was also seen with other forms of antisense oligonucleotides given bound to PESOA, and was specific for inhibition of CYP3A2 expression. (nebraska.edu)

The compounds used in the analysis were the therapeu-tic oligonucleotide mipomersen and three mipomersen analogs. (shimadzu.com)

Anatomy9

Organisms4

Chemicals and Drugs134

Analytical, Diagnostic and Therapeutic Techniques and Equipment40

Phenomena and Processes71

Disciplines and Occupations3

Technology, Industry, Agriculture6

Information Science3

Health Care3

Peptides3

ASOs7

Field of oligonucleotide1

Modulate4

Synthetic8

20212

Chemically modified1

Synthesis market7

Phosphorothioate1

Gene expression1

Molecules2

Analytical1

Biomarkers1

Pharmaceutical6

Chemotherapeutic agents2

MDM23

Proteins2

Achieve therapeutic2

Human umbilical1

Approaches4

Reagents1

Aptamers3

Cancer therapeutic1

Nanoparticles1

Potential8

Inhibitors1

Genes2

Cell4

Workflows1

Therapy1

Molecule2

Delivery2

Developments2

Diseases2

Manufacture1

Molecular4

Targets2

Chemistry1

Clinical application2

Vivo1

Medicines1

Development4

20221

Diagnostics1

Research4

Artificial1

Treatment3

Induce1

Duchenne2

Inhibition1

Analysis1