111 results about "Rna targeting" patented technology

The invention belongs to the field of medicines, particularly relates to the technical field of targeted delivery lipids, and particularly discloses an ionizable cationic lipid targeting immune cells, a composition containing the ionizable cationic lipid and application of the ionizable cationic lipid. The immune cell targeting cationic lipid provided by the invention has a structure as shown in formula (I), can be used for RNA targeting delivery, and can significantly enhance the targeting effect of mRNA drugs on immune cells. # imgabs0 #

The invention discloses a target segment for preventing and treating tomato brown fruit wrinkling virus in tobacco, double-stranded RNA (Ribonucleic Acid) and application of the target segment and the double-stranded RNA. Wherein the nucleotide sequence of the target segment is as shown in SEQ ID NO.3, or a sequence which is as shown in SEQ ID NO.3, has at least 90% of homology and has coded amino acid as shown in SEQ ID NO.4 is substituted. The invention also discloses a double-stranded RNA of a targeted interference target section, and the double-stranded RNA can significantly inhibit the proliferation of the tomato brown crinkled fruit virus in tobacco by injecting or smearing tobacco leaves, so that the double-stranded RNA can be effectively applied to prevention and control of tobacco diseases caused by the tomato brown crinkled fruit virus. The method for preventing and controlling the tomato brown crinkled fruit virus in the tobacco by using the double-stranded RNA is safe, harmless and efficient, and provides a new path for preventing and controlling plant virus diseases by using RNAi (Ribonucleic Acid Interference).

Provided are a double-stranded RNA targeting coagulation factor XI, a pharmaceutically acceptable salt thereof, or a ligand conjugate thereof。 Further provided are a pharmaceutical composition comprising the double-stranded RNA, the pharmaceutically acceptable salt thereof, or the ligand conjugate thereof, as well as a therapeutic use of the double-stranded RNA, the pharmaceutically acceptable salt thereof, or the ligand conjugate thereof.

The invention discloses a preparation method of an RNA targeted photosensitizer and an application of the RNA targeted photosensitizer in anti-tumor treatment, and belongs to the technical field of fine chemical engineering. The photosensitizer is modified on the basis of a fluorescent molecule parent, and the RNA targeted photosensitizer is constructed. The photosensitizer disclosed by the invention can be self-assembled into nanoparticles in an aqueous solution, shows good RNA targeting, effectively generates reactive oxygen species through I-type and II-type mechanisms, and shows excellent performance in the aspects of long-term fluorescence imaging in tumor cells and anti-tumor treatment in normal-oxygen and hypoxic environments; the fluorescence of the photosensitizer Se-cy is enhanced by about 230 times after RNA is added, and the fluorescence duration can be kept as long as 40 hours in in-vitro cells; and the fluorescent label can be kept for 4 days in vivo, shows excellent fluorescent durability, and has a remarkable anti-tumor effect on tumor-bearing mice.

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 angiotensinogen (AGT). Such compounds are useful in methods for reducing expression of AGT and for therapeutically treating RAAS associated diseases, disorders and / or conditions, or symptoms thereof in a subject.

The present disclosure is directed to a system 100 and a method 300 for predicting binding affinity of small molecules to Ribo-Nucleic Acid (RNA) targets The method comprises of receiving an input from a user, comprising a sequence of an RNA target, information related to a small molecule and a sub-type of the RNA target. The method further comprises of predicting a binding affinity of the small molecule to the RNA target of the sub-type, using a prediction model. Generating the prediction model comprises of, obtaining a plurality of RNA sequence based features for a plurality of sub-types of a plurality of RNA targets, and a plurality of small molecule features for a plurality of small molecules. Generating the prediction model further comprises of identifying a plurality of non-correlated features from the plurality of RNA sequence based features and the plurality of small molecule features and generating a final feature combination from the plurality of non- correlated features for each sub-type of the plurality of sub-types of the plurality of RNA targets based on a quasi-binding affinity of each of the plurality of small molecules with each sub-type of the plurality of sub-types of the plurality of RNA targets. The final feature combination is used to generate the prediction model.

The invention discloses a target segment and double-stranded RNA for preventing and controlling propagation of tomato brown crinkled fruit virus through tomato seeds and application of the target segment and the double-stranded RNA. Wherein the nucleotide sequence of the target segment is as shown in SEQ ID NO.3, or a sequence which is as shown in SEQ ID NO.3, has at least 90% of homology and has coded amino acid as shown in SEQ ID NO.4 is substituted. The invention further discloses double-stranded RNA of the target interference target section, the double-stranded RNA is used for single isolation treatment or batch treatment of germinated tomato seeds, and the infection rate of the tomato brown crinkled fruit virus on germinated tomato seedlings can be effectively reduced under the condition that the germination rate of the tomato seeds is not affected. The method for preventing and controlling propagation of the tomato brown crinkled fruit virus through the tomato seeds by using the double-stranded RNA is safe and harmless, and has a good application prospect.

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.

The present disclosure provides a universal and multifunctional platform for transcriptome modulation that utilizes RNA-guided RNA targeting activity that binds to a VI-D type CRISPR effector with RNA-guided RNA endonuclease activity that expresses a guide array of a plurality of guide RNAs. The system can be used for quantitative, reversible and large-scale multiplex gene knock-down in primary human T cells, and multiplex inhibition of consumption related genes in T cells. The system can be used for enhancing the anti-tumor activity of dysfunctional CAR T cells.

The invention discloses a time-space resolution single cell transcriptome detection method, which comprises the following steps: firstly, carrying out specific marking on RNA newly generated by cells within a specific time by using a metabolism marking technology so as to ensure that new RNA molecules can be accurately identified and tracked; then, signal amplification of a new RNA target is realized through an adjacent rolling circle amplification (PLA-RCA) technology, and the detection sensitivity is improved; and finally, realizing high-throughput analysis on the newborn RNA by using an in-situ fluorescence sequencing technology, and carrying out accurate space positioning and quantification on the newborn RNA in the single cell by the step. Through the method, time and space resolution analysis can be provided for a single cell transcriptome, so that analysis of complex biological processes such as cell differentiation, disease occurrence and cell function response is facilitated; moreover, the method has important significance for revealing a molecular mechanism in a cell biological process, and provides a new visual angle and tool for disease diagnosis and treatment.

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.

Disclosed herein are compositions comprising nanoparticles designed for CRISPR / Cas13 RNA targeting systems, specifically aimed at targeting poorly druggable, disease-driving genes in prostate cancer and COVID-19, and methods of use thereof.

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 application discloses a multiplex method for measuring nucleic acid modification enzyme activity and screening one or more variable elements of enzymatic reaction.The method includes: (i) providing a polynucleotide construct library encoding DNA / RNA target site and variable element to be tested such as nucleic acid modification enzyme variant, (ii) partitioning a single copy of the polynucleotide construct with in vitro transcription and translation (IVVT) reagent, (iii) allowing the IVVT reaction of nucleic acid modification enzyme and DNA / RNA target site to be expressed in each compartment, (iv) depending on the functionality of the nucleic acid modification enzyme encoded, single polynucleotide construct / RNA target is cracked, remains intact or otherwise modified, and (v) by sequencing to cracked, complete or modified polynucleotide construct / RNA target quantification, so as to directly identify the enzymatic activity related to each variable element and to the enzymatic activity quantification.Particularly, nucleic acid modification enzyme is CRISPR associated protein (Cas).

In one aspect, compositions are provided for the simultaneous in situ quantification and localization of RNA sequences with subcellular precision are utilized in the methods. In particular, specific hybridization of at least one probe set in a sample is followed by in situ ligation, which locks specifically circularized probe set around an RNA target sequence. Rolling circle amplification followed by fluorescently labeled detector probe hybridization, enables simultaneous in situ quantification and localization of RNA sequences with subcellular precision.

The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.

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 disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.

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 invention discloses an in-situ capture, amplification and sequencing method of an RNA (Ribonucleic Acid) target sequence, which is characterized in that a lock-type probe with a gap is designed aiming at the RNA target sequence, the 5'end and the 3 'end of the lock-type probe are respectively complementarily hybridized with the upstream and downstream of the target sequence, and a middle gap corresponds to the target sequence. CDNA of a target sequence is generated through reverse transcription, and the cDNA is connected with the 5'end of the probe through DNA ligase to form annular DNA. And after rolling circle amplification, target sequence information is obtained through in-situ sequencing. According to the method, the experimental process is simplified, the detection sensitivity and specificity are improved, the method is suitable for various sample types, and an efficient tool is provided for spatial transcriptomics research.

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.

Disclosed herein are high efficiency RNA targeting (ARNATAR) dsRNA compounds that target angiotensinogen (AGT). Such compounds are useful in methods of reducing AGT expression and therapeutically treating RAAS-related diseases, disorders and / or conditions or symptoms thereof in a subject.