Articles tagged with Target Mrna
A team of researchers has demonstrated a means by which CRISPR/Cas9 can be programmed to recognize and cleave RNA at sequence-specific target sites. This allows for direct RNA transcript detection, analysis and manipulation, paving the way for transformative studies in RNA function.
Researchers have identified a small molecule capable of interrupting the disease process in cells carrying the C9ORF72 gene, a variant associated with ALS and FTD. The compound selectively targets abnormal RNA molecules, reducing their accumulation and potentially serving as a biomarker for clinical trials.
Researchers have revealed the structure of the Cas9 complex, a key part of the CRISPR-Cas system used for genome editing. The study provides a detailed picture of the complex, enabling researchers to refine and engineer the tool to accelerate genomic research and bring it closer to treating human genetic disease.
Berkeley researchers provide detailed picture of Cas9's three-dimensional shape, showing radical change in structure upon binding to guide RNA. This breakthrough enables rational design of new and improved versions of Cas9 enzymes for basic research and genetic engineering.
Scientists at Scripps Research Institute have developed a novel method to increase the potency of RNA treatments, resulting in a 2,500-fold improvement. The breakthrough enables precise targeting of disease-causing RNAs and could lead to more effective therapeutic agents.
Researchers at Scripps Florida have developed a new method to alter RNA function in living cells by designing molecules that recognize and disable disease-associated RNAs. This approach has the potential to treat genetic disorders such as myotonic dystrophy, which can cause muscle wasting and other symptoms.
Researchers have designed a compound that shows promise as a potential therapy for fragile X syndrome, a genetic condition causing mental retardation, infertility, and memory impairment. The molecule improves RNA splicing process and minimizes repeat-associated defects in cells.
A Cold Spring Harbor Laboratory study reveals a new way in which the cell's splicing machinery recognizes splice sites, impacting current ideas on how missteps triggered by mutations can lead to diseases. The discovery affects up to 5% of all splice sites and has implications for pinpointing splicing defects underlying certain diseases.
Researchers develop miR-TRAP, a new method to directly identify microRNA targets in cells. This technique allows scientists to understand the roles microRNAs play in human development and disease, bridging a gap in the RNA field.
A Scripps Research Institute scientist has developed a novel method to design small molecule therapeutics targeting RNA, leveraging a bottom-up approach that uses information on RNA folds and internal loops. The successful designs have shown strong activity against myotonic dystrophy type 1, a common form of muscular dystrophy.
Researchers have determined the three-dimensional atomic structure of Argonaute2, a key 'gene silencer' protein involved in regulating cell activities. This discovery paves the way for understanding RNA-silencing and harnessing it to treat diseases by designing better therapeutic guide RNAs.
Researchers designed a series of small molecules that target an RNA defect causing myotonic dystrophy type 1. These compounds improve biological defects in cell culture and animal models by more than 40 percent.
Researchers at Scripps Research have identified a compound that can repair specific defects in RNA, a key step in developing treatments for incurable diseases like Huntington's. The new technology targets toxic RNA defects associated with Spinocerebellar ataxia and Kennedy disease.
A study by SUNY Downstate scientist Ilham Muslimov suggests that molecular competition in neuronal RNA transport may contribute to neurodegenerative disorders. The researchers identified RNA motifs that act as spatial codes in nerve cells, directing RNAs to dendrites and synapses.
Researchers at University of Michigan developed a new way to search for drugs that target RNA, a molecule essential to retroviruses like HIV. They successfully predicted the binding of six new small molecules to HIV's genetic material and demonstrated their efficacy in inhibiting viral replication.
Researchers successfully modified messenger RNA to override a 'red light' signal, producing a full-length protein instead. This breakthrough may aid treatment strategies for genetic disorders caused by premature stop codons.
Scientists identified a class of RNAs called CIRTs that target genetic building blocks to guide protein synthesis in nerve cell dendrites. This discovery provides clues for understanding brain disorders and highlights the potential impact of viral infections on normal cellular function.
Researchers at CSHL discovered new modes of mRNA regulation involving Ago2 and Drosha proteins, highlighting a previously unappreciated complexity in gene expression control. The study found that mRNAs can be targeted for destruction by multiple molecules, expanding our understanding of post-transcriptional events.
The study identified specific binding sites of microRNAs in C. elegans, providing a wealth of data for understanding miRNA regulation in development and disease. This breakthrough enables researchers to identify individual miRNA targets in various tissues and cell types.
Researchers at the University of Illinois have designed a small molecule that blocks an aberrant pathway associated with myotonic dystrophy type 1. The new compound, Ligand 1, binds tightly to its target, preventing the MBNL protein from binding to RNA and easing symptoms of the disease.
Researchers found that Kaposi's sarcoma-associated herpesvirus uses polyadenylation to block normal gene expression in cells. The virus' SOX protein aberrantly lengthens mRNA poly(A) tails, sending a signal to the cell that its messages are wrong and holding them back.
Biomedical engineers have developed a new probe that allows visualization of single RNA molecules within live cells, enabling scientists to study RNA's operation and interaction with binding proteins. The tool overcomes issues with fluorescent probes, allowing for hours-long imaging and distinguishing between targeted and unbound probes.
Researchers at Rutgers University have created a novel gene silencing platform called U1 Adaptor that targets RNA biosynthesis. The platform has the potential to treat diseases resistant to current RNAi approaches and can inhibit genes that do not respond to existing methods.
Researchers discovered that U1, a guiding RNA molecule, can 'slide' one base to recognize atypical splice sites, explaining the genetic error in PCH. This shift enables U1 to form stronger matches with divergent sequences.
Researchers at the University of Rochester Medical Center have identified several compounds that block unwanted RNA coupling, a key step in the disease. The discovery offers hope for developing a drug-like molecule to treat myotonic muscular dystrophy.
A joint research study by IBM and the Genome Institute of Singapore found that microRNAs control stem cell differentiation through coding regions beyond the 3'UTR, challenging previous assumptions. The discovery has implications for novel diagnostics and therapeutics.
Researchers found Ago2 necessary for normal blood cell development, but its role is independent of slicer activity. Ago2 regulates miRNA biogenesis in blood cells through translational control.
Researchers at Yale University have identified a new regulatory target for the Fragile X mental retardation protein (FMRP), which may lead to new treatments for Fragile X syndrome. The study also found implications for autism, as both conditions share common physiological pathways.
The discovery of microRNAs in the unicellular green alga Chlamydomonas reinhardtii expands our understanding of small RNA regulation and challenges existing dogma. The researchers found functional characteristics between plant and animal miRNAs, suggesting a potential role in regulating sexual reproduction.
Researchers discover miRNAs silence genes through two independent mechanisms: repression of translation and induction of mRNA degradation. This finding resolves controversy over whether miRNAs affect mRNA levels.
A University at Buffalo medicinal chemist is working to develop rules for targeting RNA, which could lead to the design of efficient compounds to inhibit specific RNA sequences. This approach has the potential to treat diseases such as cancer and genetic disorders, offering a more targeted alternative to DNA-based treatments.
Researchers at UT Southwestern discovered that microRNA miR-1 targets the mRNA of the gene Hand2, a key regulator of heart formation. This fine-tuning allows proper heart muscle development and may aid in understanding congenital heart disease.
Researchers discover small RNA molecules in plant phloem, suggesting a novel role in long-distance signaling and stress response. A new protein is identified as likely playing a key role in transporting these RNAs through the phloem.
Researchers developed a colorimetric detection capability for Nanosphere's nanoparticle-based molecular detection systems, improving the identification of genomic DNA, RNA, and protein targets. The new technology enables sensitive and specific detection without amplification procedures.
Researchers at Georgia Tech and Emory University have developed improved molecular beacons for detecting viruses and cancer cells in living cells. The beacons use fluorescent dyes and oligonucleotides to target specific genetic sequences, producing a distinct optical signal when excited by light.
Researchers at Rockefeller University have discovered that FMRP controls the fate of specific proteins in brain cells, explaining the physical, cognitive, and behavioral abnormalities characteristic of fragile X syndrome. The findings offer potential for future therapies to lessen the disease's impact.
Researchers at MGH/Harvard discovered a tiny RNA gene controlling developmental timing in animals, including fish, sea urchins, and humans. The gene is conserved across bilaterally symmetric animals but not in more primitive or plant species.
Researchers at UCSF have discovered a region in the telomerase enzyme that could be targeted to kill cancer cells and regenerate damaged cells. The discovery provides new insights into the mechanism of telomerase and its potential as a therapeutic target, as well as its role in regulating cell life span.
Scientists have identified enzymes called kinases that add phosphates to inositol, triggering the export of messenger RNA from the cell nucleus. The discovery provides insight into a previously unknown signaling pathway and its role in regulating gene expression.
Researchers link destruction of proteins to destruction of messenger RNA, a breakthrough that could lead to development of pharmaceuticals to control protein production.
A chemist at Washington University has created a molecule that mimics the behavior of ribozymes, acting as a catalyst to dismantle dangerous genetic codes involved in viral and fungal diseases, certain cancers, and HIV. The discovery shows great promise for improved drug treatments.
Scientists at UNC Chapel Hill and Bern University have developed a new RNA repair technique to block defective processing in cells' messenger RNAs. This method has shown promising results in increasing healthy protein production and could potentially treat or even cure beta thalassemia.
Chemist Benjamin Miller has devised a way to create new drugs by using metal atoms to assemble countless combinations of molecules, then selecting the best candidates through a Darwinian process. This method shifts the burden of tedious drug development work off technicians' shoulders, offering a faster and more efficient approach.
Researchers successfully infect chimpanzees with a defined HCV sequence, identifying essential elements for infection and paving the way for the development of better treatments. The availability of this infectious sequence will enable precise studies of HCV replication and inform the creation of effective vaccines or immunotherapies.