Abstract: New dosing regimens for treating muscular dystrophy in a patient suffering from Duchenne muscular dystrophy (DMD) with an antisense oligonucleotide conjugate that causes skipping of an exon in the human dystrophin gene are described. Also described is a method of treating a patient with an antisense oligomer CPP conjugate and a magnesium supplement.

Abstract: Disclosed are methods of detecting myocardial enhancer RNA levels in mammalian cardiomyocytes. In a further embodiment, a method of disrupting assembly of an enhancer DNA-Myh6 promoter-Myh7 promoter complex, is provided wherein the method comprises providing enhancer RNA to a cardiomyocyte.

Abstract: The invention provides a diagnostic kit for metastasis and invasion of breast cancer and a use of an shRNA molecule for silencing expression of human LINC01614. The shRNAs obtained by the invention can interfere with the expression of LINC01614, thereby reducing the migration and invasion ability of tumor cells, inhibiting the expression of EMT proteins, and inhibiting tumor formation and lung metastasis in an animal model in vivo. The invention provides a new solution for targeted therapy of breast cancer. Therefore, the kit for diagnosing metastasis and invasion of breast cancer and the medication for treating metastasis and invasion of breast cancer are developed. The invention provides a new way and strategy for diagnosing and treating metastasis and invasion of breast cancer.

Abstract: The present disclosure relates to compositions that upregulate the production of one or more sequences of micro-interfering ribonucleic acid (miRNA). The sequences of miRNA may be complimentary to a sequence of target messenger RNA (mRNA) that encodes for translation of a target biomolecule, and the miRNA may cause the target mRNA to be degraded or inactivated, thereby causing a decrease in bioavailability of the target biomolecule because it is degraded or inactivated by the miRNA, thereby decreasing the bioavailability of the target biomolecule within a subject that is administered the one or more compositions. In some embodiments of the present disclosure, the target biomolecule is an opioid receptor. In some embodiments of the present disclosure, the target biomolecule is one or more of the mu opioid receptor, the delta opioid receptor, the kappa opioid receptor and the nociceptin opioid receptor.

Abstract: The present invention relates to 5?-cap compounds, in particular the stabilization of RNA by such 5?-cap compounds, and provides compositions, such as pharmaceutical compositions, and cells comprising an RNA which is modified with such a 5?-cap compound, as well as methods for producing a peptide or protein of interest using the compositions or cells according to the present invention. Furthermore, the present invention provides the RNA, compositions, or cells for use in therapy, in particular for use in a method of treating a disease or disorder by protein replacement therapy, genome engineering, genetic reprogramming, and immunotherapy; a method for increasing the stability of RNA in cells; a method for increasing the expression of RNA in cells; and a method for providing an RNA with a 5?-cap structure.

Abstract: The present invention concerns an in vivo method for introducing an mRNA molecule (which is not associated with a carrier) into the cytosol of a cell(s) in a subject, by the use of photochemical internalization, wherein the photosensitising agent is a sulphonated meso-tetraphenyl chlorin, sulfonated tetraphenylporphine or a di- or tetrasulfonated aluminium phthalocyanine used in an amount of 0.0001 to 1 ?g. The method may be used to express a polypeptide in the subject. The invention is also directed to pharmaceutical compositions containing the photosensitising agents and the mRNA and uses of the molecules in therapy, e.g. to treat or prevent cancer or an infection.

Abstract: Disclosed are messenger RNA molecules and related compositions incorporating a 4?-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4?-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.

Abstract: A method for elongating telomeres of cells comprises steps of: providing physical stimulation directly or indirectly to cells; and culturing a mixture of the cells and a medium for a predetermined time, wherein providing the stimulation directly to the cells comprises applying physical stimulation to the medium containing the cells, and providing the stimulation indirectly to the cells comprises applying physical stimulation to the medium not containing the cells and then mixing the medium and the cells. The method for elongating telomeres of cells is simpler than a conventional method and is superior in terms of time, cost, efficiency, and safety. In addition, the method induces cell division and provides an anti-aging effect, in addition to simply elongating telomeres. Thereby, it is expected that the method can ameliorate and prevent not only problems caused by shortening of telomeres, but also various aging-related diseases and conditions.

Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.

Abstract: The present disclosure relates to RNAi agents, for example, double stranded RNAi agents, able to inhibit HIF-2 alpha (EPAS1) gene expression. Also disclosed are pharmaceutical compositions that include HIF-2 alpha RNAi agents and methods of use thereof. The HIF-2 alpha RNAi agents disclosed herein may be linked or conjugated to targeting ligands (such as compounds that have affinity for integrins, including alpha-v-beta-3 and alpha-v-beta-5 integrins) and pharmacokinetic (PK) enhancers, to facilitate the delivery to cells and tissues, including to clear cell renal cell carcinoma (ccRCC) cells and tumors. Delivery of compositions comprising the HIF-2 alpha RNAi agents in vivo provides for inhibition of HIF-2 alpha gene expression. The HIF-2 alpha RNAi agents can be used in methods of treatment of various diseases and disorders, including ccRCC.

Abstract: The present disclosure provides methods of treating subjects having liver disease, and methods of identifying subjects having an increased risk of developing liver disease.

Abstract: Described herein are saturating agents, AAV gene therapy vectors, and therapeutic agents, as well as methods and kits comprising the same.

Abstract: The present disclosure provides siRNA that suppresses proliferation of new coronaviruses (SARS-CoV-2). The siRNA disclosed herein includes: a sense strand; and an antisense strand. The sense strand includes a target sequence comprising 19 to 23 bases in which a base at a 5? terminal is guanine (G) or cytosine (C), and an overhang comprising 2 to 4 bases added to a 3? terminal side of the target sequence. The antisense strand includes a sequence complementary to the target sequence, and an overhang comprising 2 to 4 bases added to a 3? terminal side of the complementary sequence. Here, at least a part of the target sequence contains at least a part of a base sequence encoding a signal peptide region of a spike protein (S protein) of SARS-CoV-2.

Abstract: The present invention relates to a composition for temperature sensors including graphene oxide, DNAzyme and PNA, a temperature sensing method and a kit using the same. In the present invention, when the DNAzyme/PNA duplex is dissociated at a certain temperature, graphene oxide adsorbs PNA with excellent selectivity and irreversibility, enabling recall of temperature, to permit delayed color development in the time of need. Also, the target temperature can be easily and quickly detected with the naked eye through the color change of the colorimetric reagent, and the thermosensor is technically convenient and easy to apply, so it can be used in various biological applications. Moreover, it can be used as a barcode (on/off) system using a combination of PNA probes with various lengths, and thus can be broadly applied to sensing a diverse range of temperatures.

Abstract: Disclosed herein are CCR7 aptamers that specifically bind to and internalize into CCR7-expressing cells. The aptamers can be used as a therapeutic agent by itself or in combination with a small molecule by forming a conjugate with the small molecule. Targeted delivery of the small molecule can be achieved by such conjugates. Also disclosed are methods of treating a subject suffering from cancer by administering an effective amount of the aptamer or the aptamer-small molecule conjugate to the subject.

Abstract: The present invention relates to antisense oligonucleotides for modulating the function of a T cell, including antisense oligonucleotides that hybridise to IFN-?, granzyme, perforin 1, PD-1, PRDM1, PD-L1, CD40LG, NDFIP1, PDCD1 LG2, REL, BTLA, CD80, CD160, CD244, LAG3, TIGIT, ADORA2A & TIM-3 RNAs. In particular, the present invention relates to antisense oligonucleotides capable of inducing exon skipping of RNA. Also claimed is a method for further modifying the specificity of said T-cell by providing for a T cell receptor gene.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: SARS-CoV-2 causes pandemic COVID19. Developing an effective treatment to directly target the virus could significantly impact viral burden in those most vulnerable to the devastating effects of this virus. The invention provides antisense oligonucleotides (AOs) to target the single stranded RNA genome of the SARS-CoV-2 viruses. The administration of AOs can significantly reduce the target viral RNAs.

Abstract: Methods of alleviating or prophylactically blocking pain through the alteration of certain metabolic pathways in a subject experiencing pain or expecting to experience pain are disclosed. In particular, methods of increasing the amount of the neutral amino acid transporter ASCT2 in neurons are disclosed. Increased glutamine uptake by neurons leads to reduced extrusion of lactate and protons into the extracellular space and by extension, reduced sensitization of the neurons. Reduced sensitization of the neurons lessens the feeling of pain in the subject.

Abstract: The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.