Patent classifications
C12N2310/18
Single stranded oligonucleotides inhibiting endocytosis
The invention provides a single-stranded oligonucleotide (ssON) for use in preventing or treating an influenza virus infection in a subject, wherein the single-stranded oligonucleotide is one that inhibits endocytosis. The invention also provides a single-stranded oligonucleotide (ssON) that comprises or consists of a polynucleotide having a sequence sharing at least 60% sequence identity with a sequence of any one of SEQ ID NOs: 13-21 listed in Table 1, or a complementary sequence thereof.
Compositions and methods for genomic editing by insertion of donor polynucleotides
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 (gDNA) 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.
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.
CELL PENETRATING PROTEIN-ANTIBODY CONJUGATES AND METHODS OF USE
Provided herein are cell penetrating conjugates. The conjugates include non-cell penetrating proteins connected through a phosphorothioate nucleic acid, wherein the phosphorothioate nucleic acid enhances intracellular delivery of the non-cell penetrating proteins. Also provided are methods and kits including the conjugates provided herein.
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.
Components and methods for producing toxic RNAs in eukaryotic cells
Disclosed are components and methods for producing toxic RNAs and/or extracellular vesicles comprising the toxic RNAs in cells that are resistant to the toxic RNAs, which toxic RNAs may include toxic shRNA/siRNA and/or toxic pre-miRNA/miRNA. The disclosed components may include engineered cells that can be utilized to express the toxic RNAs, in which the engineered cells do not express one or more genes that are required for processing the toxic RNAs for RNA interference (RNAi) and/or one or more genes that are required for executing RNAi. The toxic RNAs and/or extracellular vesicles comprising the toxic RNAs may be utilized in methods for treating diseases and disorders through RNAi.
Systems, methods, and compounds for providing chaperone activity to proteins
Systems and method for providing chaperone activity to a protein-containing compound is disclosed. The method includes selecting a nucleic acid based on one or more of the nucleic acid's particular properties and a specific sequence of the nucleic acid and applying the nucleic acid to a compound comprising one or more proteins to provide chaperone activity to the compound.
ACTIVATORS OF TYPE III CAS PROTEINS
Described herein are compositions and systems comprising activators of type III accessory nucleases and methods of using these compositions and systems.
Compositions and methods for genomic editing by insertion of donor polynucleotides
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.
Shape-Responsive Nanostructures
Provided herein are single-stranded tile (SST) structures prepared from and comprising single-stranded γPNA (ss-γPNA) strands, along with methods of making an SST structure from single-stranded γPNA (ss-γPNA) strands.