Articles tagged with Oligonucleotides
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Scientists create a 'sense' DNA drug that can undo the effects of an antisense drug used to treat blood clots. The new approach reversed the antisense drug's activity and restored normal levels of prothrombin, preventing bleeding. This breakthrough demonstrates the feasibility of using oligonucleotides to target specific genes.
Researchers at Tokyo Medical and Dental University developed a novel DNA/RNA heteroduplex oligonucleotide (HDO) that significantly improves gene silencing and reduces liver dysfunction. The high potency of vitamin E-conjugated HDO results in improved delivery to the liver, leading to enhanced therapeutic effects.
MGN1703 is a single-stranded oligonucleotide-based drug that activates the human immune system without causing harmful side effects. It works by binding to toll-like receptor 9, triggering a cascade of signaling pathways that enable recognition and destruction of foreign cells.
Researchers have developed nanoparticles that can efficiently transport charge-neutral oligonucleotide analogs into cells, enabling them to retain biological activity. This breakthrough delivery approach has the potential to treat various therapeutic targets.
The review article explores the therapeutic advantages of phosphorothioate groups in oligonucleotides, enabling novel gene expression-regulation therapies. Novel applications include microRNA and long non-coding RNA targets and decoy oligonucleotides.
The article presents new consensus guidelines for toxicity testing of oligonucleotide drugs, considering their unique chemical and biological characteristics. The guidelines aim to design scientifically valid and predictive toxicity studies.
A novel RNAi-based therapy has shown promising results in treating anemia by stimulating the liver to produce erythropoietin (EPO). The treatment targets the EGLN1 gene, leading to increased EPO and hemoglobin levels. This breakthrough could provide a safer alternative to current recombinant human EPO treatments.
Researchers have developed a promising new approach to editing gene transcripts, which uses targeted oligonucleotide drugs. This technique has already shown promise in treating diseases such as Duchenne Muscular Dystrophy and spinal muscular atrophy.
A group of industry and regulatory scientists released consensus recommendations for assessing the safety pharmacology evaluations of oligonucleotide-based therapeutics. The guidelines emphasize the importance of safety pharmacology studies to evaluate cardiovascular function effects.
Researchers use antisense oligonucleotides to skip over the mutated exon causing Huntington's disease, preventing formation of toxic protein fragments. The innovative therapeutic strategy is a proof-of-concept for treating complex diseases.
A new study demonstrates the effectiveness of antisense drugs in targeting a wide range of tissues and organs, including liver, kidney, lung, muscle, and peripheral nerves. The findings suggest that antisense therapeutics have broad therapeutic potential for various disease indications.
A novel machine learning-based approach predicts hepatotoxic potential of antisense oligonucleotides based on chemical sequence. The method achieved 74% accuracy in predicting toxicity and was used to redesign a therapeutic oligonucleotide with reduced potential for liver harm.
Researchers have identified key regions in the dengue virus genome that can be targeted to prevent replication. Artificial microRNAs were constructed to specifically target these sites, leading to effective inhibition of viral replication in humans. This innovative approach offers new hope for treating millions affected by dengue fever.
A new method for manufacturing short, single-stranded DNA molecules has been developed by researchers at Karolinska Institutet and Harvard University. This technique can produce large amounts of DNA copies cheaply using bacteria, improving the quality and scalability of DNA fragment production.
Gene Signal's aganirsen shows promise in treating wet age-related macular degeneration and ischemic retinopathy by inhibiting neovascular growth. Clinical studies are scheduled to begin during the second quarter of 2012.
Myotonic dystrophy is caused by a mutation that causes toxic RNA to accumulate in cells. Antisense oligonucleotides have been shown to be effective in cell culture and mice by degrading the toxic RNA. The treatment will need to be refined for systemic delivery to patients with myotonic dystrophy.
A new DNA testing method, MEMO, has been developed to detect trace mutant DNA sequences with improved sensitivity. The technique uses 3′-modified oligonucleotides to block normal gene extension while allowing mutated gene extension, increasing detection sensitivity.
Antigene therapy utilizes a DNA-based drug that attaches directly to specific DNA sequences and is activated by light energy, effectively silencing targeted genes. This approach may offer new hope for treating diseases currently incurable or having limited success.
Researchers developed DNA-like compounds that effectively inhibit cells responsible for systemic lupus erythematosus, a potentially groundbreaking treatment. The findings demonstrate the anti-inflammatory effects of class R inhibitory oligonucleotides and could lead to new therapies.
The development of siRNA drugs is hindered by a strong immune response that can cause toxic side effects. Researchers are exploring mechanisms for inducing this response and strategies for minimizing its effects.
Researchers delivered antisense oligonucleotides through cerebrospinal fluid to treat neurodegenerative diseases like ALS. The therapy effectively modulated protein levels and slowed disease progression in rats.
Researchers found that glucocorticoids (GCs) alter the activity of bone-forming and bone-degrading cells, leading to delayed bone formation and resulting in GC-induced osteoporosis. Antisense oligonucleotides can be delivered through cerebrospinal fluid to treat neurodegenerative diseases like ALS.
Researchers develop system to study small RNAs using siRNA inhibitors, providing unprecedented insight into RNA interference and microRNA functions. The technique enables rapid discovery of hundreds of microRNAs' regulatory roles, with significant implications for genetic studies.
Researchers at UNC used antisense oligonucleotides to target RNA splicing pathways implicated in cancer and genetic diseases. The technique successfully sensitized cancer cells to chemotherapy and radiation, offering a potential breakthrough for targeted therapy.