C12N2310/18

Long noncoding RNAs and cell reprogramming and differentiation

Long noncoding RNAs (lncRNAs) are identified that enhance pluripotency reprogramming of somatic cells as well as differentiation of pluripotent cells. Induced pluripotent stem (iPS) cell generation from somatic cells leads to the upregulation and downregulation of identified lncRNAs. The modulation of these lncRNAs are capable of enhancing pluripotency of somatic cells as well as enhancing differentiation of a pluripotent cell.

NEUROPROTECTIVE MOLECULES AND METHODS OF TREATING NEUROLOGICAL DISORDERS AND INDUCING STRESS GRANULES
20200010832 · 2020-01-09 ·

Provided herein are neuroprotective molecules containing a sequence of 25-35 contiguous nucleotides between nucleotide 1 and nucleotide 50 of a mature human tRNA and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure, the at least four contiguous guanosine-containing nucleotides are located at the 5 end of the neuroprotective molecule, and the neuroprotective molecule contains at least one deoxyribonucleotide. Also provided are neuroprotective molecules containing a sequence of 25-35 contiguous nucleotides between nucleotide 1 and nucleotide 50 of a mature human tRNA.sup.CYS; and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure and the at least four contiguous guanosine-containing nucleotides are located at the 5 end of the neuroprotective molecule.

Heteromultivalent Nucleic Acid Scaffolds and Uses Thereof

The present disclosure relates to heteromultivalent nucleic acid scaffolds and peptides, and to compositions comprising the scaffolds and peptides, and to methods for preparing and using the scaffolds and peptides. The present disclosure also relates to use of the scaffolds, peptides, and compositions for preventing or disrupting interactions between molecules, in particular protein-protein interactions, in particular for the treatment or prevention of disease, in particular cancer, such as breast cancer.

Nucleic acid oligomers and uses therefor

Disclosed are nucleic acid oligomer compounds and to their use in compositions and methods for inhibiting proliferation, survival or viability of cancer cells including prostate, lung, pancreatic, breast, cervical and bone cancer cells.

AN ENGINEERED MULTI-COMPONENT SYSTEM FOR IDENTIFICATION AND CHARACTERISATION OF T-CELL RECEPTORS AND T-CELL ANTIGENS

The present invention relates to A multicomponent system wherein a first component is an engineered antigen-presenting cell (eAPC) designated component A and a second component is a genetic donor vector, designated component C, for delivery of one or more ORFs encoding an analyte antigen-presenting complex (aAPX) and/or an analyte antigenic molecule (aAM), wherein component A a. Lacks endogenous surface expression of at least one family of aAPX and/or aAM and b. Contains at least two genomic receiver GO sites, designated component B and component D, each for integration of at least one ORF encoding at least one aAPX and/or aAM, and component C is matched to a component B, and wherein component C is designed to deliver c. A single ORF encoding at least one aAPX and/or aAM or d. Two or more ORF encoding at least one aAPX and/or aAM, wherein the genomic receiver sites B and D are synthetic constructs designed for re-combinase mediated exchange (RMCE).

PROTECTING RNAS FROM DEGRADATION USING ENGINEERED VIRAL RNAS

This invention is in the field of molecular biology, gene expression, functional genomics, and bioinformatics and relates to novel RNA and related structures and methods of use thereof that enables modulation of gene expression and preservation of particular transcriptome targets. The invention contemplates various applications of RNA sequences derived from the genomic RNA of flaviviruses (FVs) and the application of such features in combination with heterologous sequences.

Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules

Provided herein are neuroprotective molecules containing a sequence of 25-35 contiguous nucleotides between nucleotide 1 and nucleotide 50 of a mature human tRNA and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure, the at least four contiguous guanosine-containing nucleotides are located at the 5 end of the neuroprotective molecule, and the neuroprotective molecule contains at least one deoxyribonucleotide. Also provided are neuroprotective molecules containing a sequence of 25-35 contiguous between nucleotide 1 and nucleotide 50 of a mature human tRNA.sup.CYS; and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure and the at least four contiguous guanosine-containing nucleotides are located at the 5 end of the neuroprotective molecule.

COMPOSITIONS AND METHODS FOR GENOMIC EDITING BY INSERTION OF DONOR POLYNUCLEOTIDES
20240167060 · 2024-05-23 ·

The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA molecule in a cell. In some embodiments the present disclosure provides donor polynucleotides comprising two strands capable of correcting a mutation that causes Glycogen Storage Disease 1a.

Expression Inhibitor of Inflammation Promoting Factor, Screening Method for Active Ingredient Thereof, Expression Cassette Useful for Said Method, Diagnostic Agent and Diagnosis Method

Provided are an expression inhibitor of an inflammation promoting factor based on the discovery of a new factor influencing the expression amount/level of an inflammation promoting factor, and a development tool therefor. Provided are also a diagnostic agent and a diagnosis method for immune diseases, inflammatory diseases, painful conditions and similar. More specifically provided are: an expression inhibitor of an inflammation promoting factor containing at least one kind of inhibitor selected from the group consisting of RBMS2 expression inhibitor and RBMS2 function inhibitor; a screening method using as an indicator the expression or the function of RBMS2; an expression cassette useful for said method; as well as a diagnostic agent containing a product detection agent for RBMS2 gene expression and disease detection method using as an indicator RBMS2 gene expression amount/level.

Compositions and methods for treating cancer, inhibiting cell proliferation, and inducing cell death

Quadruplex-forming guanine-rich nucleic acid sequences are useful in compositions and methods for inhibiting cellular growth and proliferation and inducing cell death. Compositions for treating a patient are provided, including (i) a safe and effective amount of a sequence having at least 80% nucleic acid identity with a sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO:3, SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6, SEQ ID NO: 7, SEQ ID NO: 8, SEQ ID NO: 9, SEQ ID NO:10, SEQ ID NO:11, SEQ ID NO:12 and combinations thereof, and (ii) a carrier, wherein the isolated oligonucleotide forms at least one quadruplex.