Patent classifications
A61K31/7125
METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
Disclosed herein are methods for decreasing AlAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis. Disclosed herein are methods for decreasing A1AT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of liver disease, such as, A1ATD associated liver disease, and pulmonary disease, such as, A1ATD associated pulmonary disease in an individual in need thereof. Methods for inhibiting AlAT mRNA and protein expression can also be used as a prophylactic treatment to prevent individuals at risk for developing a liver disease, such as, A1ATD associated liver disease and pulmonary disease, such as, A1ATD associated pulmonary disease.
NUCLEIC ACID MOLECULES FOR REDUCTION OF PAPD5 OR PAPD7 MRNA FOR TREATING HEPATITIS B INFECTION
The present invention relates to a method for identifying a compound that prevents, ameliorates and/or inhibits a hepatitis virus (HBV) infection, wherein a compound that reduces the expression and/or activity of PAP associated domain containing 5 (PAPD5) and/or PAP associated domain containing 7 (PAPD7) is identified as a compound that prevents, ameliorates and/or inhibits a BV infection. The invention also provides for inhibitors of PAPD5 or PAPD7 for use in treating and/or preventing a HBV infection; as well as a combined preparation comprising an inhibitor of PAPD5 and an inhibitor of PAPD7 for simultaneous or sequential use in the treatment or prevention of a HBV infection. Also comprised in the present invention is a pharmaceutical composition for use in the treatment and/or prevention of a HBV infection, and a method for monitoring the therapeutic success during the treatment of a HBV infection.
NUCLEIC ACID MOLECULES FOR REDUCTION OF PAPD5 OR PAPD7 MRNA FOR TREATING HEPATITIS B INFECTION
The present invention relates to a method for identifying a compound that prevents, ameliorates and/or inhibits a hepatitis virus (HBV) infection, wherein a compound that reduces the expression and/or activity of PAP associated domain containing 5 (PAPD5) and/or PAP associated domain containing 7 (PAPD7) is identified as a compound that prevents, ameliorates and/or inhibits a BV infection. The invention also provides for inhibitors of PAPD5 or PAPD7 for use in treating and/or preventing a HBV infection; as well as a combined preparation comprising an inhibitor of PAPD5 and an inhibitor of PAPD7 for simultaneous or sequential use in the treatment or prevention of a HBV infection. Also comprised in the present invention is a pharmaceutical composition for use in the treatment and/or prevention of a HBV infection, and a method for monitoring the therapeutic success during the treatment of a HBV infection.
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.
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.
PROTEINS AND NUCLEIC ACIDS FOR EHRLICHIA DIAGNOSIS AND VACCINATION
Methods and compositions for diagnosing and vaccinating against Ehrlichia canis and Ehrlichia chaffeensis are provided.
PROTEINS AND NUCLEIC ACIDS FOR EHRLICHIA DIAGNOSIS AND VACCINATION
Methods and compositions for diagnosing and vaccinating against Ehrlichia canis and Ehrlichia chaffeensis are provided.
CONJUGATE GROUP AND CONJUGATE
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.
PHARMACEUTICAL COMPOSITION COMPRISING ORGANOID AND ANTI-INFLAMMATORY AGENT FOR PREVENTING OR TREATING INFLAMMATORY BOWEL DISEASE
The present disclosure relates to a pharmaceutical composition for preventing or treating inflammatory bowel disease, comprising an intestinal organoid and a TNFα inhibitor.
The combination use of the intestinal organoid and TNFα inhibitor according to the present disclosure can alleviate symptoms of inflammatory bowel disease such as weight loss, diarrhea, and bloody stool, and can inhibit fibrosis of the intestinal mucosa due to inflammation. In particular, when the intestinal organoid and the anti-inflammatory TNFα inhibitor are used in combination, the therapeutic effect is significantly superior to that of multiple administrations of the TNFα inhibitor, and thus it can be usefully used as an agent for preventing or treating inflammatory bowel disease.
PHARMACEUTICAL COMPOSITION COMPRISING ORGANOID AND ANTI-INFLAMMATORY AGENT FOR PREVENTING OR TREATING INFLAMMATORY BOWEL DISEASE
The present disclosure relates to a pharmaceutical composition for preventing or treating inflammatory bowel disease, comprising an intestinal organoid and a TNFα inhibitor.
The combination use of the intestinal organoid and TNFα inhibitor according to the present disclosure can alleviate symptoms of inflammatory bowel disease such as weight loss, diarrhea, and bloody stool, and can inhibit fibrosis of the intestinal mucosa due to inflammation. In particular, when the intestinal organoid and the anti-inflammatory TNFα inhibitor are used in combination, the therapeutic effect is significantly superior to that of multiple administrations of the TNFα inhibitor, and thus it can be usefully used as an agent for preventing or treating inflammatory bowel disease.