C12N15/1131

METHODS AND COMPOSITIONS OF RNA NANOSTRUCTURES FOR REPLICATION AND SUB-GENOMIC EXPRESSION BY RNA-DIRECTED RNA POLYMERASE
20230227830 · 2023-07-20 ·

The present invention is directed to methods and compositions of RNA nanostructures for replication and/or subgenomic expression of gene modulating single-stranded RNA by RNA-directed RNA polymerase-like proteins and the use of such nanostructures for use in a variety of organisms.

Aptamers for targeting HPV16-positive tumor cells

A composition for binding to human papillomavirus type 16 (HPV16)-positive tumor cells, the composition including a DNA aptamer. The DNA aptamer includes one of SEQ ID NO: 1 and SEQ ID NO: 2.

Inhibitor for use for preventing and/or treating an infection with hepatitis B virus

An inhibitor for use for preventing and/or treating an infection with hepatitis B virus (HBV) and an in vitro screening method for the identification of a candidate compound suitable for preventing and/or treating an infection with hepatitis B virus is provided.

INHIBITION OF POLYOMAVIRUS REPLICATION

The invention relates to antisense molecules and methods for modulating splicing of polyomavirus T antigen pre-mRNA. In one aspect the invention relates to an antisense oligonucleotide 12 to 30, preferably 17, 18, 19 or 20 to 30 nucleobases in length which comprises a sequence that is the reverse complement of a contiguous stretch of at least 12 nucleobases of a polyomavirus T-antigen pre-mRNA and which antisense oligonucleotide can modulate splicing of said T-antigen pre-mRNA in a cell.

DELIVERY OF GENE EXPRESSION MODULATING AGENTS FOR THERAPY AGAINST CANCER AND VIRAL INFECTION
20230212566 · 2023-07-06 ·

Methods and agents that target nanog or Oct4 expression or activity for treating or preventing cancer are disclosed. Alternative methods involve diagnosing cancer stage or type by identifying presence of cancer cells expressing nanog or Oct4. Also, disclosed are method of treating coronavirus infection that involves administering antiviral knockdown agents, such as oligonucleotide-based inhibitors.

COMPOSITIONS AND METHODS OF INDUCING RNAI OR TYPE I IFN COMPETENT CELLS AND USES THEREOF
20230212581 · 2023-07-06 ·

Provided is a double stranded RNA (dsRNA) compound comprising a guide strand and a passenger strand, the guide strand and the passenger strand each having a length of at least 300 basepairs (bp), the guide strand comprising a segment complementary to a target nucleic acid sequence of a target gene transcript. Also provided are methods of silencing a target gene transcript in a vertebrate cell or subject, of treating a pathogen infection in a subject, and of reducing replication or infectivity of a pathogen infection in the vertebrate cell or subject, respectively, comprising administering to the subject or cell a dsRNA compound, vector, conjugate or composition herein disclosed.

CONJUGATE GROUP AND CONJUGATE
20230210882 · 2023-07-06 · ·

The present application relates to a novel conjugate group linkable to a compound (such as a therapeutic compound), for use in directing the compound to a target in the body. The conjugate group disclosed herein can enable an expression-inhibitory oligonucleotide (such as a RNAi reagent) to target liver cells to regulate gene expression. The conjugate group disclosed herein has a variety of applications when linked to expression-inhibitory oligonucleotides, comprising applications in therapy, diagnosis, target verification, and genome development. A composition comprising the conjugate group disclosed herein can mediate expression of target nucleic acid sequences in liver cells (such as hepatocytes) when linked to expression-inhibitory oligonucleotides, and can be used for treatment of diseases or disorders responding to the gene expression or activity of cells, tissues, or organisms.

Reagents for treatment of hepatitis B virus (HBV) infection and use thereof
11535851 · 2022-12-27 · ·

This disclosure relates to RNA interference (RNAi) reagents for treatment of hepatitis B virus (HBV) infection, compositions comprising same, and use thereof to treat individuals infected with HBV. The reagents are artificial miRNA (shmiRNA) used alone or in combination with additional shmiRNA or shRNA.

NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING SAME, AND PREPARATION METHOD AND USE
20220395526 · 2022-12-15 ·

Provided are a siRNA for inhibiting the expression of hepatitis B virus gene, and a pharmaceutical composition and conjugate containing the siRNA. Each nucleotide in the siRNA is independently a modified nucleotide. The siRNA comprises a sense strand and an antisense strand. The sense strand of the siRNA comprises a nucleotide sequence 1 having the same length and no more than three nucleotides different from the nucleotide sequence shown in SEQ ID NO: 155, and the antisense strand of the siRNA comprises a nucleotide sequence 2 having the same length and no more than three nucleotides different from the nucleotide sequence shown in SEQ ID NO: 156.