Gene expression by antisense oligonucleotide. Medical search. Frequent questions

REHOVOT and RAMAT GAN , Israel , Nov. 21, 2023 /PRNewswire/ -- Skip Therapeutics (" Skip "), developer of computational platform for design of Antisense Oligonucleotide (ASO) based therapies and Sheba Medical Center (" Sheba "), one of the leading medical centers in the world, announced today that they have entered a collaboration for the development of ASO-based treatments for rare genetic diseases. (ktla.com)
Recent advancements in RNA-therapeutics offer an expanded molecular toolkit that can be deployed rapidly and at relatively low costs to restore protein function of mutated genes. (ktla.com)
Afterwards, I joined the gene therapy laboratory at Royal Holloway, and since then I contributed to the development of novel gene therapy agents and antisense therapeutics for the treatment of muscular dystrophies and other muscle conditions. (royalholloway.ac.uk)
In 2013, I re-joined the gene therapy laboratory at Royal Holloway as project manager to work on the optimization of gene therapies and antisense therapeutics for a number of muscle diseases. (royalholloway.ac.uk)
We have solid collaborations with national and international academics and with Industry partners to develop gene therapy reagents, antisense therapeutics and pharmacological agents that have potential for clinical translation. (royalholloway.ac.uk)
This review examines the potential of RNA therapeutics, focusing on antisense oligonucleotide modification of pre-mRNA splicing, methods for pre-mRNA trans-splicing, and the isoform- and allele-specific applications of RNA interference. (ox.ac.uk)
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the first quarter of 2022 and provided business updates. (financialcontent.com)
Our primary focus is to develop synthetic antisense oligonucleotide based therapeutics targeting specific non-coding RNAs as a revolutionary pharmacological tool fighting cardiovascular diseases. (mhh.de)

They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA guides. (frontiersin.org)
There are currently several products containing RNA on the market, and many are under development, among them mRNA, antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), small activating RNAs (saRNAs), micro-RNAs (mi-RNAs), RNA aptamers and RNA guides. (frontiersin.org)
Recent studies have revealed the importance of long noncoding RNAs (lncRNAs) as tissue-specific regulators of gene expression. (nature.com)
Endogenous antisense RNAs function as regulators of gene expression by a variety of mechanisms. (genomicglossaries.com)
Synthetic antisense RNAs are used to effect the functioning of specific genes for investigative or therapeutic purposes. (genomicglossaries.com)
Small noncoding RNAs called microRNAs (miRNA) negatively regulate gene expression by sequence-specific targeting of protein-coding transcripts [ 6 ]. (researchsquare.com)
This can be done by chemically modifying the oligonucleotides like CpG, anti-gene, small non-coding RNAs, etc. (artofhealingcancer.com)
MicroRNAs (miRNAs) are small noncoding RNAs (~22 nucleotides in length) that regulate gene expression post-transcriptionally by imperfect binding to the 3′ untranslated region of target mRNAs in a wide variety of cell types. (biomedcentral.com)
Non-coding RNAs are novel therapeutic targets as master regulators of gene expression by controlling many physiologic and pathologic processes. (mhh.de)

Therapeutic tiny or short modified antisense oligonucleotides (Tiny ASOs) can block gene expression or modulate splice-switching! (biosyn.com)
ASOs can be used to manipulate the splicing process and restore function of a mutated gene, by promoting exclusion of the mutated region from the final RNA transcript or by restoring normal splicing where it has been lost. (ktla.com)
Skip's ASOs aim to restore function of mutated genes in genetic disorders by modulating splicing or by controlling gene expression levels. (ktla.com)
Indeed, functional studies in animals, mainly using antisense oligonucleotides (ASOs) termed antagomirs, have demonstrated that targeting miRNAs can alter evoked and spontaneous seizures and neuropathological outcomes including neuronal loss and gliosis [ 11 ]. (researchsquare.com)

Phosphoryl guanidine oligo-2′-O-methylribonucleotides in neutral therapeutic oligonucleotides enable enhanced cell penetration! (biosyn.com)
Therapeutic Oligonucleotides target the mRNA and change gene expression (proteins). (artofhealingcancer.com)
The aim of this project is to identify therapeutic oligonucleotides (i.e. antisense and aptamers) that can selectively target these fusion proteins. (pasteur.fr)

Since RNA-binding proteins (RBPs) are key players in the post-transcriptional regulation of gene expression precise knowledge of their binding sites is critical for determining their molecular function and for understanding their roles in cell development and disease. (biosyn.com)
More than half of all human genes are alternatively spliced, so it is expected that alternative pre- mRNA splicing will be responsible for the identification of more than 90 percent of all human proteins. (genomicglossaries.com)
The gene loci coding for each of the DAG complex proteins is located outside the X chromosomes. (medscape.com)
Oligonucleotides bind the complementary sequence and inhibit the synthesis of specific proteins by different mechanisms. (artofhealingcancer.com)
These are Oligonucleotides which cuts the expression of proteins with in a cell. (artofhealingcancer.com)
These oligonucleotides bind in the 5 to 3 directions with mRNA and do not allow it to translate into proteins. (artofhealingcancer.com)
They will not allow RNA to form proteins, or they may destroy the RNA molecule thus downregulating the target gene. (artofhealingcancer.com)
They bind the complementary RNA that will be either degraded in the cell or will not be able to translate into proteins thus affecting the expression of oncogenes in the cell. (artofhealingcancer.com)
The oligonucleotide sequence binds the mRNA in the cell and prevents the synthesis of specific proteins from it. (artofhealingcancer.com)
Elucidating the mechanism of over and under expression of proteins is critical in developing a better understanding of cancer. (biomedcentral.com)
Multiple techniques are used to examine differential expression of proteins in cells and assess changes in protein expression in response to therapies such as radiation. (biomedcentral.com)
To reduce the risk of analyzing cell line- pathway can be divided into distinct stages based on the specific features, we used several representative cell lines for recombination status of the Ig genes and on the expression each of four major stages in B cell development: pro-B, pre-B, pattern of surface markers and the presence of intracellular proteins [1- 6]. (lu.se)

A first RNA molecule comprises a double-stranded sequence, which includes a guide strand sequence that is complementary to a portion of an mRNA transcript encoded by the target gene. (genomeweb.com)
Preferably, the second guide strand sequence comprises one or more bases that are mismatched with a nucleic acid sequence of the mRNA transcript encoded by the target gene. (genomeweb.com)
The method is adaptable for high-throughput screening methods and is suited for identifying polynucleotides that can catalyze cleavage of target mRNA and/or act on target mRNA through and antisense or RNAi mechanism. (genomeweb.com)
In the study we have evaluated the efficiency of antisense oligonucleotides (AS-ODNs) against TF selected from computational prediction of TF mRNA structure. (oldcitypublishing.com)
In conclusion, we compared inhibition of TF gene expression in different cancer cell lines and found that all effective AS-ODNs were located in the translated region of TF mRNA. (oldcitypublishing.com)
Because alternative RNA splicing allows different types of mRNA molecules to be created from a single gene, it generates the diversity of protein function and structure that is essential to complex organisms. (genomicglossaries.com)
High- fidelity mRNA amplification for gene profiling. (genomicglossaries.com)
RNA-like oligonucleotides that are complementary to a portion of a target mRNA molecule. (genomicglossaries.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)
Highly specific and effective gene silencing of any disease can be achieved by an accurate knowledge of the target mRNA sequence and rational design of its complementary antisense agents for the downregulation of its protein message. (genomicglossaries.com)
The use of oligonucleotides that are complementary to our target mRNA in order to reduce the expression of some specific gene in a cell is called antisense therapy. (artofhealingcancer.com)
modalities because oligonucleotides can easily be modified, they have a high affinity to bind targeted mRNA, they are highly specific, and have no side effects. (artofhealingcancer.com)
This prevention may result when oligonucleotides cause inhibition of the translation process, they may prevent the spicing of exons, or they inhibit the transport of mRNA from the nucleus into the cytoplasm which is important for translation to occur. (artofhealingcancer.com)
Reduced expression can be caused by protein inactivation, mRNA instability, or reduced transcription. (biomedcentral.com)
The antisense oligonucleotides dramatically reduced survival of multiple myeloma cells and decreased human IRF4 messenger RNA (mRNA) and protein expression, as well as lowered expression of the IRF4 target gene MYC. (scienceboard.net)
The cells were sensitive to the treatment with significant reductions in cell viability and IRF4 mRNA expression. (scienceboard.net)
In xenograft mouse models, the treatment was well tolerated in myeloma tumor-bearing mice and significantly reduced tumor growth and IRF4 mRNA expression in tumor tissues in a dose-dependent manner after two to six weeks of treatments. (scienceboard.net)
Most antisense oligos had no effect on mRNA. (epigenie.com)
RNA-based strategies offer a series of novel therapeutic applications, including altered processing of the target pre-mRNA transcript, reprogramming of genetic defects through mRNA repair, and the targeted silencing of allele- or isoform-specific gene transcripts. (ox.ac.uk)

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)

and introducing at least one siRNA that is complementary to the siRNA tag of the transfected genetic construct to inhibit the expression of the target gene. (genomeweb.com)

We look forward to propelling gene therapy forward as a result of this exciting collaboration. (ktla.com)
ASHEVILLE, North Carolina - Epidermolysis bullosa (EB), a heterogeneous congenital condition of skin fragility, received its first FDA-approved gene therapy only a few months ago, but accelerated progress across multiple treatment strategies predicts additional important and perhaps dramatic further progress, according to a prominent EB researcher. (medscape.com)
In addition to cell therapies and gene therapies, Mellerio discussed progress in three additional areas: gene editing, protein therapy, and drug repurposing. (medscape.com)
The success is supporting further work on this approach but has also been an inspiration to other gene therapies, including a topical gene therapy recently approved in the United States. (medscape.com)
Perhaps less advanced but still promising, protein therapy, gene editing, and repurposing of existing therapies are all approaches that are moving forward. (medscape.com)
These studies demonstrate that PESDA microbubbles in the absence of ultrasound can significantly improve the biologic effectiveness of targeted gene therapy. (nebraska.edu)
Antisense oligonucleotide therapy plays a vital role in cancer and genetic disorders. (artofhealingcancer.com)
This therapy involves the use of oligonucleotides that are complementary to specific target DNA or RNA in the nucleus. (artofhealingcancer.com)
So, oligonucleotides used for antisense therapy should be modified in such a way that they have high stability and low side effects. (artofhealingcancer.com)
The use of oligonucleotides for the treatment of cancer patients is found to be a very effective therapy. (artofhealingcancer.com)
Antisense therapy is effective in the sense that it can distinguish between the normal and mutated oncogenes in cells and targets only mutated oncogenes without downregulating the expression of normal genes. (artofhealingcancer.com)
I collaborated on the development of a gene therapy AAV vector for Oculopharyngeal Muscular Dystrophy (OPMD), a rare muscle disease. (royalholloway.ac.uk)
I worked within the Unite DMD consortium to complete the preclinical development of an AAV-microdystrophin gene therapy that is now in clinical trial in France and UK. (royalholloway.ac.uk)
We use gene therapy technologies to target molecular defects in pre-clinical models of rare neuromuscular diseases such as Duchenne muscular dystrophy (DMD), Oculopharyngeal muscular dystrophy (OPMD) and Facioscapulohumeral muscular dystrophy (FSHD). (royalholloway.ac.uk)
Pharmacological agents that can improve the dystrophic pathology and that can have additive or synergistic effects when combined with gene therapy applications. (royalholloway.ac.uk)
Gene Therapy. (royalholloway.ac.uk)
Modulating the expression of disease genes with RNA-based therapy. (ox.ac.uk)
Conventional gene therapy has focused largely on gene replacement in target cells. (ox.ac.uk)
Qalsody TM is an antisense oligonucleotide therapy that targets expression of the superoxide dismutase 1 gene. (patexia.com)
On May 16, 2022, the Company expects to present preclinical data supporting its approach to treating ADOA with STK-002 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, at 10:30am ET. (financialcontent.com)
The research will involve the MIND Institute's interventional genetics team, which includes internationally recognized experts in mouse models for therapeutic development and in gene therapy, including the use of the gene editing tool CRISPR . (ucdavis.edu)
Antisense oligonucleotides are DNA or RNA strands that are used in antisense therapy. (biologyonline.com)
Oligomers are also important for use in antisense therapy. (biologyonline.com)
With the advances in BMT and gene therapy, patients now have a better likelihood of developing a functional immune system in a previously lethal genetic disease. (medscape.com)
[ 5 ] Nusinersin is an antisense therapy. (medscape.com)
A second approach was also studied, [ 6 ] with a single-dose gene replacement therapy for SMA in which the SMA gene is applied with a viral vector. (medscape.com)
it showed an improvement in achieving motor milestones in these children after gene therapy. (medscape.com)

Locked Nucleic Acid in PCR primers, qPCR probes, and other types of oligonucleotides is soluble in water and standard buffers as well as follows Watson-Crick base-pairing rules. (sigmaaldrich.com)

SYNGAP1 encephalopathy is an autosomal dominant genetic disorder caused by haploinsufficiency of the SynGAP protein, usually due to the presence of a heterozygous protein-truncating loss-of-function variation on the SYNGAP1 gene. (wikipedia.org)
The use of antisense oligonucleotides to up-regulate the expression of SynGAP protein is currently being researched. (wikipedia.org)
Deep RNA-DNA, RNA-protein interaction studies, and phenotype rescue analyses reveal that LETR1 is a nuclear trans-acting lncRNA modulating, via key epigenetic factors, the expression of essential target genes, including KLF4 and SEMA3C , governing the growth and migratory ability of LECs. (nature.com)
The dystrophin gene is located on the short arm of chromosome X near the p21 locus and codes for the large protein Dp427, which contains 3685 amino acids. (medscape.com)
Attenuation of matrix protein secretion by antisense oligodeoxynucleotides to the cyclin kinase inhibitor p21(Waf1/Cip1). (biomedcentral.com)
ADNP syndrome is a complex neurodevelopmental condition caused by a mutation or change in the ADNP (Activity Dependent Neuroprotective Protein) gene, which affects brain development, connectivity and function. (ucdavis.edu)
Gene knockdown is essential in understanding the function of a particular protein. (biologyonline.com)
Aptamers can be used as drug delivery systems for the treatment of GB and antisense oligonucleotides can silence essential genes involved in fusion protein expression. (pasteur.fr)
Diagnosis is suggested clinically and is confirmed by genetic testing or analysis of the protein product (dystrophin) of the mutated gene. (msdmanuals.com)

MicroRNAs perform important roles in the post-transcriptional regulation of gene expression. (researchsquare.com)
Alternative approaches targeting RNA have the potential to circumvent some of these difficulties, particularly as the active therapeutic molecules are usually short oligonucleotides and the target gene transcript is under endogenous regulation. (ox.ac.uk)
In essence, oligomers are involved in the regulation of gene expression (e.g. microRNA). (biologyonline.com)

My initial work was focused on developing antisense reagents for exon skipping of Duchenne muscular dystrophy. (royalholloway.ac.uk)

They readily bind to complementary oligonucleotides or to nucleic acids, forming a duplex. (biologyonline.com)

In cell line T508 were obtained moderate effects in inhibition of TF gene expression of 30% by AS-4. (oldcitypublishing.com)
Inhibition of Xfoxi1a activities by injection of antisense oligonucleotides leads to suppression of non-CNS ectodermal markers (e.g. keratin ) and expansion of the anterior expression domain of the CNS marker Sox2 . (biologists.com)
In rats, it took 4mg/kg of AON to inhibit CYP3A2 expression when administered unbound to PESDA, while a dose of only 0.06 mg/kg of the same AON produced equivalent inhibition when given bound to PESDA microbubbles. (nebraska.edu)
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)
Drug-gene Interaction Screens Coupled to Tumor Data Analyses Identify the Most Clinically Relevant Cancer Vulnerabilities Driving Sensitivity to PARP Inhibition. (cdc.gov)

Published clinical studies of cell therapies and gene therapies date back at least 15 years, according to a review by Mellerio on why developments are starting to move so quickly. (medscape.com)

By employing microRNA expression profiling and functional knockdown studies on human embryonic stem cell-derived cardiomyocytes, the authors identified miR-363 as an upstream negative regulator of left ventricular specification transcription factor HAND1 . (biomedcentral.com)

Multiple platforms for gene editing have been described with the goal of simply excising pathogenic mutations or antisense oligonucleotides for sustained or permanent control of EB expression. (medscape.com)
[4] Mutations in the SOD1 gene are the most frequent among more than forty genes associated with ALS. (patexia.com)
They are caused by mutations of the dystrophin gene, the largest known human gene, at the Xp21.2 locus. (msdmanuals.com)
SCID results from mutations in any of more than 15 known genes. (medscape.com)

Functional analyses of ectopically expressed copies of the variant miRNA precursors demonstrate loss of function, gain of function, and altered expression levels. (genomeweb.com)
To this end, the authors performed miRNA expression profiling in undifferentiated hESCs and CMs at day 8 and day 14 after differentiation. (biomedcentral.com)

T m of duplex between an oligonucleotide and its complementary sequence. (sigmaaldrich.com)

Because transcription factors (such as IRF4) are difficult to target with traditional small-molecule strategies, researchers from the University of California (UC) San Diego School of Medicine and Ionis Pharmaceuticals used an antisense oligonucleotide-based inhibitory platform to reduce the stability of IRF4 transcripts for the purpose of disrupting cancer metabolism. (scienceboard.net)

Incorporating Locked Nucleic Acid into oligonucleotides increases thermal duplex stability 2 and improves the specificity of oligonucleotide hybridization to target sequences. (sigmaaldrich.com)
Specifically, Locked Nucleic Acid oligonucleotides can be designed to address traditionally problematic target sequences, such as AT- or GC-rich regions. (sigmaaldrich.com)
The oligonucleotides are of specific sequences complementary to the target RNA or DNA sequence. (artofhealingcancer.com)
We performed northern blot analyses for the guide RNA truncations and found increased levels of expression for the longer tracrRNA sequences, suggesting that improved target cleavage was at least partially due to higher sgRNA expression or stability ( Fig. 1c ). (cdc.gov)

Targeted AON delivery could selectively inhibit gene expression (exp). (nebraska.edu)
The researchers tested a panel of human-specific antisense oligonucleotide agents targeting IRF4 in vitro and profiled their effects on multiple myeloma cell viability and IRF4 expression to determine a candidate that selectively inhibits human IRF4. (scienceboard.net)

Furthermore, dosage-dependent antisense oligonucleotide knock-down of dync1h1 revealed outer segment abnormalities in the absence of overt inner segment polarity and trafficking defects. (nih.gov)
Oligonucleotides are used in treatments for gene defects and Metabolic disorders. (artofhealingcancer.com)

Aptamers (from the Latin aptus ("- fit") and Greek meros ("-part") that are oligonucleotides (since other aptamers can be peptide molecules) are created usually from a large random sequence pool and used mainly for research and clinical applications (e.g. as macromolecular drugs). (biologyonline.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)

In particular, the FANTOM (Functional Annotation of the Mammalian Genome) consortium pioneered the discovery of the noncoding RNA world by providing, through Cap Analysis of Gene Expression (CAGE-Seq), the first evidence that large portions of our genome are transcribed, producing a multitude of sense and antisense transcripts 12 . (nature.com)
Sequencing as well as functional studies using antisense oligonucleotides, indicate important roles for microRNAs during the development of epilepsy through targeting transcripts involved in neuronal structure, gliosis and inflammation. (researchsquare.com)
Importantly, they found that treatment reduced the expression of IRF4 target genes, as well as other microenvironment-responsive and cell cycle regulatory transcripts but spared normal hematopoietic cells. (scienceboard.net)

Therefore, this complex regulatory network requires precise control of gene expression patterns at both transcriptional and post-transcriptional levels in order to ensure proper maturation, differentiation, and formation of blood and lymphatic vessels. (nature.com)

Deafness gene screening based on a multilevel cascaded BPNN model. (cdc.gov)

So, there is no chance of multiple complementary sites for oligonucleotides in the genome and hence no harm to normal cells. (artofhealingcancer.com)
Whole genome bisulfite sequencing (WGBS) revealed a few modest (~10%) changes occurring in CpG shores and large scale (300bp) methylation differences occurring primarily over several repeated gene families. (epigenie.com)
An Integrated Approach of Learning Genetic Networks From Genome-Wide Gene Expression Data Using Gaussian Graphical Model and Monte Carlo Method. (cdc.gov)

Antisense oligonucleotide gapmers containing phosphoryl guanidine groups reverse MDR1-mediated multiple drug resistance of tumor cells. (yale.edu)

This and other drugs available for the treatment of different cancer types usually target the cancer-promoting genes in the cell (Sharad, 2019). (artofhealingcancer.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)

A number of post-transcriptional mechanisms fine-tune the gene expression landscape. (researchsquare.com)
The researchers noted that one benefit of antisense oligonucleotides is that they can be delivered into cells by free update with natural endocytic mechanisms, which enables rapid gene expression modulation compared to alternative gene delivery strategies. (scienceboard.net)

Oligonucleotides are short DNA or RNA molecules. (biologyonline.com)

Antisense downregulation of mutant huntingtin in a cell model. (biomedcentral.com)

The patent, its abstract states, relates to "duplex oligonucleotide complexes [that] can be administered to a cell, tissue or organism to silence a target gene without the aid of a transfection reagent. (genomeweb.com)
January 21, 2021 -- Using an engineered DNA therapeutic agent to silence transcription factor genes reduced myeloma stem cell abundance and increased the survival of mice bearing human myeloma, according to preclinical study data published in Cell Stem Cell on January 20. (scienceboard.net)

Most disorders are the result of a mutation in a single gene and have a deterministic genetic cause. (ktla.com)
STK-001 is a proprietary antisense oligonucleotide (ASO) being developed by Stoke as the first potential new medicine to address the genetic cause of Dravet syndrome. (yahoo.com)
Х-зчеплений рецесивний Genetic disorders determined by a single gene (Mendelian disorders) are easiest to analyze and the most well understood. (msdmanuals.com)

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)
Secondly, we tested the ability of AON bound to PESDA to modulate gene expression in the liver of adult male rats by testing the changes in efficacy, potency, and specificity of AON which inhibit cytochrome P450 3A2 (CYP3A2) expression. (nebraska.edu)
The investigators checked more than 1000 pharmaceutical compounds in cell culture and found that the beta-2 adrenoreceptor can modulate the expression of synuclein. (medscape.com)

During development, the blood vascular system arises from endothelial cell progenitors that differentiate from mesodermal cells, mostly through the expression of the transcription factor (TF) ETV2. (nature.com)
Activation of the VEGFA/VEGFR2 signaling and expression of blood vascular endothelial cell (BEC) markers, such as NRP1 and EphrinB2, further differentiate these precursor cells into BECs, which then form the hierarchical network of blood vessels 4 . (nature.com)

Subsequent antisense oligonucleotide-knockdown transcriptomic profiling of two LEC- and two BEC-specific lncRNAs identifies LETR1 as a critical gatekeeper of the global LEC transcriptome. (nature.com)
Subsequent differentiation allows for rear- analysis of a set of mouse B lineage cell lines rep- rangements of the Ig light-chain (IgL) genes that replace the resenting defined stages of B cell development us- surrogate light-chain genes on the surface of the B cell [8]. (lu.se)

The recent identification of mammalian circadian clock genes now makes it possible to examine time zone adjustments from the perspective of molecular events within the suprachiasmatic nucleus (SCN), the principal circadian oscillator. (jneurosci.org)
These results reveal the potential for dissociation of mPer and mCry expression within the central oscillator during circadian resetting and a differential molecular response of the clock during advance and delay resetting. (jneurosci.org)
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)
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)
Furthermore, the increasing adoption of oligonucleotides in molecular diagnostics and clinical applications contributes to market growth. (mordorintelligence.com)
In molecular genetics, they refer to a short sequence synthesised to match a regionwhere a mutation is known to occur, and then used as a probe (oligonucleotide probes). (biologyonline.com)

A diagnosis is established following the identification of a heterozygous pathogenic (or likely pathogenic) point mutation of the SYNGAP1 gene (present in approximately 89% of patients), a micro deletion of chromosome 6 incorporating SYNGAP1 (approximately 11% of patients), or a balanced translocation disrupting SYNGAP1. (wikipedia.org)

MicroRNAs regulate target gene expression post-transcriptionally in a myriad of cell types and play critical roles in diverse physiological and pathological processes, including cardiomyocyte development, differentiation, and regeneration. (biomedcentral.com)

Individual miRNAs are capable of regulating gene networks by interacting with multiple targets or by suppressing transcriptional controllers [ 6 , 8 ]. (researchsquare.com)
Over-expression of miR-22 has been reported to protect in models of brain injury and additional targets of miR-22 have been identified [ 17 , 18 ]. (researchsquare.com)
The team then showed that injecting the antisense oligos into zygotes decreased MERVL targets later in development. (epigenie.com)

The duplex oligonucleotide complexes of the disclosure include a conjugate moiety that facilitates delivery to a cell, tissue, or organism. (genomeweb.com)
The presence of a single base mismatch has a greater destabilizing effect on the duplex formation between an Locked Nucleic Acid oligonucleotide and its target than with a native-state DNA oligonucleotide. (sigmaaldrich.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)
Moreover, various companies are investing in oligonucleotide synthesis, which is expected to increase market growth over the forecast period. (mordorintelligence.com)

Thus, oligonucleotides stop the metastasis and invasion of tumors (Jansen & Zangemeister-Wittke, 2002). (artofhealingcancer.com)

A biological process that is crucial for gene expression in most animal life, including humans. (genomicglossaries.com)

methods for reducing the expression level of a target gene. (genomeweb.com)
The invention relates to a method for inhibiting the expression of a target gene in a eukaryotic cell," the patent's abstract states. (genomeweb.com)
In addition, the improved hybridization of the PCR primer or qPCR probe to its target may increase the melting temperature (T m ) by up to 8 °C per Locked Nucleic Acid monomer substitution in medium salt conditions compared to native-state DNA oligonucleotides 4 ( Table 1 ). (sigmaaldrich.com)
Thus, downregulating the expression of target genes. (artofhealingcancer.com)
When oligonucleotide-based drugs are given to the cancer patient, the drugs target the specific mutated genes in cancerous cells. (artofhealingcancer.com)

The Gene Medicine Laboratory for Rare Diseases is focused on finding new treatments for muscle pathologies. (royalholloway.ac.uk)
Patients who have Duchenne dystrophy should be offered prednisone or deflazacort and sometimes exon-skipping treatments using antisense oligonucleotides. (msdmanuals.com)

The team then carried out functional experiments in an embryonic stem cell system in which they used antisense LNA-containing oligonucleotides to interfere with specific tRFs and analyzed gene expression. (epigenie.com)

Morpholino oligomers are used in gene expression modification. (biologyonline.com)

It is one of the top single-gene causes of autism. (ucdavis.edu)

Expression of various miRNAs is dysregulated in experimental models of status epilepticus and in resected brain tissue from patients with drug-resistant temporal lobe epilepsy [ 11-13 ]. (researchsquare.com)

Table 1 Increasing the number of Locked Nucleic Acid bases in an oligonucleotide increases the T m . (sigmaaldrich.com)
The ability of oligonucleotides to discriminate between alleles via SNPs is greatly enhanced by the incorporation of Locked Nucleic Acid bases 6-8 ( Figure 2 ). (sigmaaldrich.com)
Shorter oligonucleotides incorporating Locked Nucleic Acid bases can be used at the same temperatures as longer native-state DNA oligonucleotides. (sigmaaldrich.com)
Due to Locked Nucleic Acid's improved hybridization characteristics with accompanied increase in T m , Locked Nucleic Acid oligonucleotides can be synthesized to be shorter, which overcomes certain design limitations that occur with native-state DNA oligonucleotides. (sigmaaldrich.com)
With Locked Nucleic Acid oligonucleotides, the selective placement of Locked Nucleic Acid bases facilitates the optimal design of highly-specific, shorter oligonucleotides that perform well, even at lengths of only 13 to 20 bases. (sigmaaldrich.com)
Also, the design of oligonucleotides for targeting difficult SNPs, such as the relatively stable G:T mismatch, is greatly facilitated by Locked Nucleic Acid. (sigmaaldrich.com)

Pharmaceutical biology antisense, antisense DNA, antisense oligonucleotides. (genomicglossaries.com)
and cell biology techniques for studying cell signalling, gene expression, etc. (bioethics.ac.uk)

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)

The following protocol was used to determine the mechanism for the reduced expression of an antiapoptotic factor, survivin, in normal tissues in response to radiation and the defect in cancer cells that prevents this reduction. (biomedcentral.com)

Anatomy20

Organisms16

Diseases7

Chemicals and Drugs95

Analytical, Diagnostic and Therapeutic Techniques and Equipment42

Phenomena and Processes72

Disciplines and Occupations3

Technology, Industry, Agriculture1

Information Science7

Health Care3

Therapeutics8

RNAs9

ASOs4

Therapeutic oligonucleotides3

Proteins12

MRNA19

Short strands of synthetic1

SiRNA1

Therapy27

Types of oligonucleotides1

Protein9

Regulation3

Reagents1

Nucleic acids1

Inhibition5

Therapies1

MicroRNA1

Mutations4

MiRNA2

Complementary sequence1

Metabolism1

Sequences4

Selectively2

Defects2

Aptamers1

20221

Transcripts3

Control of gene expression1

Deafness1

Genome3

Phosphoryl1

Cancer-promo1

Synthetic1

Mechanisms2

Molecules1

Downregulation1

Silence2

Genetic3

Modulate3

Endothelial2

Subsequent2

Molecular6

Pathogenic1

MicroRNAs1

Targets3

Duplex2

Synthesis3

20021

Humans1

Target5

Treatments2

Interfere1

Modification1

Single gene1

MiRNAs1

Locked Nucl6

Biology2

20212

Mechanism1