A61K31/7115

Methods for reducing LRRK2 expression

Provided herein are methods for decreasing LRRK2 mRNA expression. Such methods are useful to ameliorate LRRK2 associated diseases. Such LRRK2 associated diseases include Parkinson's Disease, including non-LRRK2 mediated Parkinson's Disease.

Methods for reducing LRRK2 expression

Provided herein are methods for decreasing LRRK2 mRNA expression. Such methods are useful to ameliorate LRRK2 associated diseases. Such LRRK2 associated diseases include Parkinson's Disease, including non-LRRK2 mediated Parkinson's Disease.

Single-stranded oligonucleotide

Provided is a single-stranded oligonucleotide that is capable of controlling a target gene with high efficiency and can be easily produced. The single-stranded oligonucleotide is represented by the formula X-L-Y wherein X and Y hybridize by a first nucleotide sequence portion and a second nucleotide sequence portion. X is composed of 7 to 100 nucleotides, contains at least one modified nucleotide, and has a first nucleotide sequence that is capable of hybridizing with a second oligonucleotide and contains at least four contiguous nucleotides recognized by RNase H. Y is composed of 4 to 100 nucleotides, and has a second nucleotide sequence that is capable of hybridizing with a second oligonucleotide and contains at least one ribonucleotide. At least one of nucleotide sequence X and nucleotide sequence Y has an antisense sequence capable of hybridizing with a target RNA. L is a group derived from a third oligonucleotide that is degraded under physiological conditions.

Use of SRSF3 agents for the treatment and/or prevention of neurological conditions, cancer, bacterial infections or viral infections
11530258 · 2022-12-20 · ·

The present description relates to the use of a SRSF3 agent for regulating the function of a myeloid cell, such as a microglial cell and/or monocyte, for treating neurological conditions, cancers, bacterial infections and viral infections wherein the SRSF3 agent inhibits expression or function of SRSF3.

Use of SRSF3 agents for the treatment and/or prevention of neurological conditions, cancer, bacterial infections or viral infections
11530258 · 2022-12-20 · ·

The present description relates to the use of a SRSF3 agent for regulating the function of a myeloid cell, such as a microglial cell and/or monocyte, for treating neurological conditions, cancers, bacterial infections and viral infections wherein the SRSF3 agent inhibits expression or function of SRSF3.

CELLULAR MOLECULAR THERANOSTICS NANOPROBE SYSTEMS AND METHODS
20220395587 · 2022-12-15 ·

A nanoprobe system for in vivo use comprises a plasmonic-active nanoparticle and a molecular probe system. The molecular probe system comprises an oligonucleotide capable of forming a stem-loop configuration, having a first end and a second end, wherein the oligonucleotide is immobilized to the plasmonic-active nanoparticle at the first end and labeled with a Raman reporter at the second end, a placeholder strand at least partially bound to the oligonucleotide, and an attachment mechanism for attachment to a cell membrane.

CFTR MRNA compositions and related methods and uses

The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.

CFTR MRNA compositions and related methods and uses

The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.

COMPOSITION FOR DETERMINATION OF CELL-MEDIATED IMMUNE RESPONSIVENESS
20220373538 · 2022-11-24 ·

The present invention relates to a composition comprising (i) a first substance which is capable to stimulate T cells, (ii) a second substance which is capable to stimulate NK cells (natural killer cells), and (iii) lipopolysaccharide (LPS) and wherein the second substance is a double stranded nucleic acid, single stranded nucleic acid, unmethylated CpG oligodeoxynucleotide, TLR agonist except lipopolysaccharide (LPS), arabinoxylan (BioBran® MGN-3), an immunoglobulin, a murine cytomegalovirus (MCMV)-encoded protein, CCL5 (chemokine (C—C motif) ligand 5), a UL-16-binding protein (ULBP), CD48, CD70, CD155, CD112, Necl-1, B7-H6, ICAM-1, RAE-1 (retinoic acid early inducible 1), 1160, Mult1 and/or hemagglutinin, to a method for measuring, determining and/or detecting the status of cell-mediated immune responsiveness of a subject, to a kit comprising the composition according to the invention, to the use of the composition for measuring, determining and/or detecting of cell-mediated immunity (CMI) and/or for detecting, diagnosing, monitoring an immunosuppression condition in a subject.

COMPOSITION FOR DETERMINATION OF CELL-MEDIATED IMMUNE RESPONSIVENESS
20220373538 · 2022-11-24 ·

The present invention relates to a composition comprising (i) a first substance which is capable to stimulate T cells, (ii) a second substance which is capable to stimulate NK cells (natural killer cells), and (iii) lipopolysaccharide (LPS) and wherein the second substance is a double stranded nucleic acid, single stranded nucleic acid, unmethylated CpG oligodeoxynucleotide, TLR agonist except lipopolysaccharide (LPS), arabinoxylan (BioBran® MGN-3), an immunoglobulin, a murine cytomegalovirus (MCMV)-encoded protein, CCL5 (chemokine (C—C motif) ligand 5), a UL-16-binding protein (ULBP), CD48, CD70, CD155, CD112, Necl-1, B7-H6, ICAM-1, RAE-1 (retinoic acid early inducible 1), 1160, Mult1 and/or hemagglutinin, to a method for measuring, determining and/or detecting the status of cell-mediated immune responsiveness of a subject, to a kit comprising the composition according to the invention, to the use of the composition for measuring, determining and/or detecting of cell-mediated immunity (CMI) and/or for detecting, diagnosing, monitoring an immunosuppression condition in a subject.