78 results about "Rna targeting" patented technology

The present disclosure relates to methods of identifying RNA targets of RNA binding proteins. In aspects, the disclosure relates to a method of identifying RNA molecules bound by RNA binding proteins. Some embodiments of the present disclosure relate to a method that can definitively identify direct RNA-target interactions with targeted proteins without the requirement for immunoprecipitation or gel extraction. In some embodiments, the method may include combining multiple antibodies in the same sample.

Disclosed herein are Advanced RNA Targeting (ARNATAR) dsRNA compounds targeting Inhibin βE (INHBE). Such compounds are useful in methods for reducing expression of INHBE and for therapeutically treating INHBE associated diseases, disorders and / or conditions, or symptoms thereof in a subject.

The invention relates to the technical field of agricultural biology, in particular to application of a neuropeptide gene CpILP6 in relieving diapause of codling moths. After siRNA is microinjected to silence CpILP6 in a targeted manner, the diapause state of CpILP6 is changed and tends to a normal development phenotype; the research result of the invention provides a theoretical basis for defining the regulation function of the neuropeptide gene CpILP6 in the diapause induction and maintenance process of codling moth.

The present application relates to isolated novel CRISPR / Cas13 proteins, a gene editing systems based thereon, and a method for using said proteins for RNA level gene editing. Provided are non-naturally occurring or engineered RNA targeting systems, and said systems each have a novel Cas13 effector protein that targets RNA and at least one type of guide molecule.

Disclosed are methods, systems, and computer readable media for predicting interactions for RNA sequences, including long noncoding RNA (IncRNA), which can be used, for example, as biomarkers. In an example method of predicting one or more binding sites between a ribonucleotide (RNA) sequence and a RNA binding protein, the method includes at a computing platform including at least one processor and memory: preparing sequence data for a plurality of overlapping RNA segments derived from a target sequence; inputting the sequence data for the plurality of overlapping RNA segments into a machine learning model trained to predict a probability of an interaction between a RNA sequence and a RNA binding protein occurring in a RNA sequence; outputting from the machine learning model one or more segments in the sequence data for the plurality of overlapping RNA segments falling above a probability threshold indicative of a region of interaction between a RNA sequence and a RNA binding protein; and predicting one or more binding sites between a RNA sequence and a RNA binding protein in the sequence data for the plurality of overlapping RNA segments derived from the target sequence based on the output from the model.

This disclosure relates to a nucleic acid comprising a double stranded RNA molecule comprising sense and antisense strands and further comprising a single stranded DNA molecule covalently linked to at least the 5′ end of either the sense or antisense RNA part of the molecule wherein the double stranded inhibitory RNA targets genes associated with cardiovascular disease in the treatment hypercholesterolemia and diseases associated with hypercholesterolemia such as cardiovascular disease.

Disclosed herein are advanced RNA targeting (ARNATAR) oligomeric compounds, which are elaborately designed compounds, for the inhibition of gene expression by the RISC pathway. Such compounds are useful in methods for reducing the expression of certain genes, where many genes are associated with a variety of diseases and disorders.

A method for the precise cellular destruction of an oncogenic non-coding RNA with a RNA-binding small molecule conjugated with bleomycin A5 is described. The method affords reversal of phenotype. Bleomycin A5 was coupled to an RNA-binding molecule that selectively binds the microRNA-96 hairpin precursor (pri-miR-96). By coupling of bleomycin A5's free amine to the RNA-binding molecule, its affinity for binding to pri-miR-96 is >100-fold stronger than to DNA. The conjugate compound selectively cleaves pri-miR-96 in triple negative breast cancer (TNBC) cells. Selective cleavage of pri-miR-96 enhances expression of FOXO1 protein, a pro-apoptotic transcription factor that miR-96 silences, and triggers apoptosis in TNBC cells. No effects were observed in healthy breast epithelial cells. This method provides programmable control for targeting RNA through the selection of an RNA-binding molecule / bleomycin A5 conjugate and provides a facile method of mapping the cellular binding sites of an RNA-binding molecule.

The invention discloses an RNA (Ribonucleic Acid) target region enrichment method based on a circular probe, and belongs to the technical field of biomolecule detection and gene sequencing. The method comprises the following steps: designing an annular probe containing an anchoring sequence, a UMI sequence and a universal primer sequence (at least one anchoring sequence 5'end is phosphorylated and modified); the method comprises the following steps: hybridizing a probe with target RNA (Ribonucleic Acid), enriching a hybridization compound through oligo d (T) magnetic beads, cyclizing the probe through DNA ligase, and carrying out PCR (Polymerase Chain Reaction) amplification to obtain an enriched product. Reverse transcription and extension steps are not needed, a solid-phase substrate is not needed, the experimental process is simplified, and the operation time is shortened; through combination of specific probe design and magnetic bead enrichment, high-selectivity enrichment of target RNA is realized, the method is especially suitable for differential expression analysis of samples with low expression quantity, and the template recovery rate and the effective data proportion are effectively improved.

The present invention provides use of lncRNA XR_595534.2 in the preparation of a medicine for the treatment or prevention of chronic pain. In the present invention, trigeminal neuralgia induced by chronic constriction injury of infraorbital nerve in rats is used as a pain model. A specifically and differentially highly expressed long-chain non-coding RNA gene lncRNA XR_595534.2 is screened in the model. An interfering RNA targeting lncRNA XR_595534.2 is provided for disease treatment. The present invention discovers for the first time that lncRNA XR_595534.2, which is specifically and differentially highly expressed in a pain model, has significantly reduced expression by stereotactic injection of an interfering RNA to relieve the pain behavior, and is useful in the preparation of a medicine for the treatment or prevention of trigeminal neuralgia, neuropathic pain, migraine and cancer pain where lncRNA-XR595534.2 is a target.

The present invention relates to a dual-target double-stranded RNA for treating hepatitis B, and a pharmaceutical composition. The dual-target double-stranded RNA comprises a first double-stranded RNA and a second double-stranded RNA, wherein one of the first double-stranded RNA and the second double-stranded RNA targets the hepatitis B virus (HBV) life cycle, and the other targets a host immune target PD-L1. The double-stranded RNA can simultaneously target the dual targets of HBV and host immune PD-L1, which can inhibit HBV replication while activating human immunity, and is therefore expected to bring about a clinical functional cure.

The application discloses a kind of polyamine, polyhydroxy ionizable cationic lipids, compositions comprising and purposes, specifically discloses a kind of cationic lipids shown in formula (I). The cationic lipids provided by the application can be used as RNA targeted delivery, can significantly enhance the spleen targeting of mRNA drug.

The embodiments disclosed herein utilize RNA targeting effectors to provide a robust CRISPR-based diagnostic for hemorrhagic fever virus applications. Embodiments disclosed herein can differentiate between hemorrhagic fever viruses that present with similar symptoms, as well as between strains of a hemorrhagic fever virus.

The present disclosure relates to the technical field of targeted delivery lipids, in particular to a cationic polymer, a lipid composition comprising the same and applications thereof. The cationic polymer provided by the present disclosure has a structure shown in formula (I), and the lipid nanoparticles prepared therefrom can be used for RNA targeted delivery, and can significantly enhance the targeting effect of mRNA drugs on liver or spleen.

The embodiments disclosed herein utilized RNA targeting effectors to provide a robust CRISPR-based diagnostic with attomolar sensitivity. Embodiments disclosed herein can detect broth DNA and RNA with comparable levels of sensitivity and can differentiate targets from non-targets based on single base pair differences. Moreover, the embodiments disclosed herein can be prepared in freeze-dried format for convenient distribution and point-of-care (POC) applications. Such embodiments are useful in multiple scenarios in human health including, for example, viral detection, bacterial strain typing, sensitive genotyping, and detection of disease-associated cell free DNA.

The invention relates to the technical field of biomedical detection, and discloses a system for accurately detecting background signal subtraction of free RNA (Ribonucleic Acid) for early diagnosis of lung cancer. Comprising a sample collection module used for obtaining whole blood of a detected sample; a base signal determination module for determining a base signal level based on the whole blood; the measured value recording module is used for recording the measured value of the differentiated cancer tissue specific RNA transcript in the plasma; the machine learning subtraction module is used for carrying out signal subtraction processing on the basic signal level and the measured value through a random forest machine learning method; and the artificial selection module is used for carrying out qualitative selection on the cancerous genome region after the signal deduction processing, and realizing background signal deduction of free RNA in the non-small cell lung cancer pathological sample and accurate verification of the cancerous genome region in combination with a selection result corresponding to the qualitative selection so as to assist in early diagnosis of lung cancer. Background signals are efficiently reduced, and tissue-specific RNA targets are accurately screened.

Compositions, kits, and methods are provided for validating patient-specific oligonuclotide assays using spike-in controls. The compositions comprise multiple sets of spike-in oligos, each set including a unique reference sequence and near-neighbor sequences with at least one substitution, insertion, or deletion. Near-neighbor concentrations are less than the reference and collectively span at least four orders of magnitude (e.g., 1:10 to 1:10,000); all sets have the same number of near-neighbors and concentration ratios. Spike-in sequences are unrelated to human sequences and are complementary to synthesis-control oligos at known concentrations. Kits further include synthesis-control oligos and patient-specific oligos for human DNA or RNA targets. Methods involve spiking a sample, co-synthesizing control and patient-specific oligos, assaying by PCR or sequencing, and validating limit of detection and dynamic range by comparing observed counts to known ratios. Applications include ctDNA and cfRNA testing.

The invention belongs to the technical field of molecular biological detection, and particularly relates to application of lysine in LAMP (loop-mediated isothermal amplification) or preparation of LAMP products. In a conventional LAMP reaction system containing a template, a primer, a buffer solution and an enzyme, lysine is innovatively introduced as a reaction synergist, it is found that non-specific amplification can be effectively inhibited, the false positive rate is remarkably reduced, meanwhile, the detection sensitivity is improved by 27.5%, and the amplification efficiency is improved by 30.4%. The method has good universality, can be suitable for detection of DNA and RNA targets at the same time, and provides a reliable technical scheme for rapid and accurate clinical diagnosis.

Taq DNA polymerase mutants exhibiting reverse transcriptase activity compared to wild type polymerase were engineered, characterized, and selected via polymerase chain reactions visualized via electrophoresis on agarose gels. Initial screening was followed up with probe-based qualitative, real-time PCR (qPCR) with a typical reverse transcription cycling protocol to detect specified ribonucleic acid (RNA) target sequences. The engineered variants can render robust cDNA from RNA target substrates and amplify that cDNA under standard reaction conditions without the assistance of added reverse transcriptase enzymes.

The application relates to a gene editing nanorobot and a preparation method and application thereof, the preparation method comprising the following steps: S1, assembling an effector protein of a CRISPR / Cas system and a guide RNA to form a complex, and crosslinking the complex to form an RNP nanocluster; S2, preparing a hybrid vesicle by using a liposome and a biologically derived vesicle; S3, loading the RNP nanocluster into the hybrid vesicle; and S4, coupling activated glucose oxidase to the surface of the hybrid vesicle to obtain the gene editing nanorobot. The gene editing nanorobot is prepared by using the preparation method. The application is applied to targeted delivery in tumor cell gene editing, RNA target regulation or gene expression regulation. The gene editing nanorobot can load a gene editing system of the effector protein of the CRISPR / Cas system, has environmental responsiveness and autonomous motion capability, and can effectively improve the delivery efficiency and application performance in gene editing.

The application provides a medicine for treating an immune checkpoint inhibitor-resistant tumor, comprising a polyunsaturated fatty acid, an ACLY inhibitor and an immune checkpoint inhibitor. The polyunsaturated fatty acid comprises one or more of omega-3 polyunsaturated fatty acids and omega-6 polyunsaturated fatty acids. The ACLY inhibitor is selected from one or more of the following: Bempedoic acid, SB 204990, NDI-091143, BMS-303141, small interfering RNA targeting the ACLY gene and short hairpin RNA targeting the ACLY gene. The medicine has good treatment effect on the immune checkpoint inhibitor-resistant tumor.

The present disclosure provides compounds of the formulae herein (e.g., Formulae (I) or (II)), and pharmaceutically acceptable salts thereof, which are degrader compounds of Quiescin Sulfhydryl Oxidase 1 (QSOX1) mRNA. The present disclosure also provides pharmaceutical compositions and kits comprising the compounds, or pharmaceutically acceptable salts thereof, and methods of treating or preventing diseases. Related compounds and methods useful in probing RNA targets and studying molecular recognition patterns between RNA and ligands are described.

The invention discloses a method for rapidly and accurately cutting RNA (Ribonucleic Acid) based on MfAgo nuclease, which belongs to the technical field of molecular biology and comprises the following steps: (1) designing and synthesizing gDNA complementary with a RNA target sequence cutting site, and phosphorylating the 5'end of the gDNA; (2) incubating MfAgo nuclease and the gDNA obtained in the step (1) in an enzyme digestion buffer solution to form an MfAgo-gDNA compound; (3) adding an RNA (Ribonucleic Acid) target into the MfAgo-gDNA compound obtained in the step (2); reacting at 55-75 DEG C for more than 15 minutes to cut the RNA target; the method has accurate single base recognition capability and is extremely sensitive to the mismatching of the bases of the guide and the Target, and the cutting efficiency can be obviously reduced or even completely disappear when the mismatching of the guide and the Target exists, so that the method has extremely low miss rate and is expected to be further used as a gene editing tool.

The invention belongs to the technical field of virus detection, and particularly relates to a method for marking a positive single-stranded RNA (Ribonucleic Acid) virus replication site in a plant living cell, which comprises the following steps: by taking pAB1677 plasmid as a template, amplifying dCas13BT3 by utilizing primers shown in SEQ ID NO.1 and SEQ ID NO.2, and connecting the dCas13BT3 into a fluorescent protein expression vector to obtain a recombinant vector; preparing gRNA of a target positive single-stranded RNA virus negative strand; and co-transferring the recombinant vector, the gRNA targeting the negative strand of the positive single-stranded RNA virus and the RNA of the positive single-stranded RNA virus into a plant, culturing, and observing the fluorescent protein expression position which is the replication site of the positive single-stranded RNA virus. According to the present invention, by designing the gRNA targeting the PnMV negative chain RNA and using the fusion of dCas13BT3 and the fluorescent protein, the fluorescence signal is accurately enriched in the virus replication complex, such that the background noise problem caused by the positive chain RNA targeting is effectively overcome;

Taq DNA polymerase mutants exhibiting reverse transcriptase activity compared to wild type polymerase were engineered, characterized, and selected via polymerase chain reactions visualized via electrophoresis on agarose gels. Initial screening was followed up with probe-based qualitative, real-time PCR (qPCR) with a typical reverse transcription cycling protocol to detect specified ribonucleic acid (RNA) target sequences. The engineered variants can render robust cDNA from RNA target substrates and amplify that cDNA under standard reaction conditions without the assistance of added reverse transcriptase enzymes.

Aspects of the disclosure relate to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of an INHBA and / or inhibit (e.g., decrease) expression of an mutated ACVR1 gene and / or promote (e.g., increase) expression of wild-type ACVR1 protein in muscle cells, bone cells or connective tissues.

This invention covers processes that amplify multiple DNA and RNA targets, solving the “multiplexed PCR problem”, as well as supporting isothermal amplification and delivering other capabilities in multiplexed molecular analysis. It uses inventive DNA analogs.

The present invention features RNA polynucleotide constructs comprising a sequence that targets Huntingtin mRNA, polynucleotide constructs comprising a sequence encoding such RNA constructs, and primary microRNA scaffolds. The sequences that comprise and / or encode targeted Huntingtin mRNA may be used, for example, to inhibit the expression of mutant HTT and / or to treat Huntington's disease.

This invention provides an optimized method for enhancing self-amplifying RNA expression and an enhanced self-amplifying RNA. Specifically, it provides an optimized method for enhancing self-amplifying RNA expression, which involves codon optimization. The optimized region is a specific region of the alphavirus non-structural protein sequence of the self-amplifying RNA, where the alphavirus non-structural protein is nsP1234. It also provides a method for preparing the enhanced self-amplifying RNA, an enhanced self-amplifying RNA, and a plasmid. This application improves the expression efficiency of the self-amplifying RNA target gene by designing and constructing codon sequence optimization for certain regions of these non-structural proteins.