A61K31/713

COMBINATION THERAPIES FOR THE TREATMENT OF CANCER
20230008508 · 2023-01-12 ·

Disclosed are compositions comprising a glycolysis inhibitor and a HDAC inhibitor and methods for treatment of cancer using said compositions.

COMBINATION THERAPIES FOR THE TREATMENT OF CANCER
20230008508 · 2023-01-12 ·

Disclosed are compositions comprising a glycolysis inhibitor and a HDAC inhibitor and methods for treatment of cancer using said compositions.

LEMD3 antagonizes TGF-beta-driven Smad2/3 transcription in a stiffness-dependent fashion in both the nucleus and cytosol

Methods and compositions for modulating transforming growth factor-beta (TGFβ) biological activity in a vertebrate subject in need thereof. The methods involve administering to the vertebrate subject an effective amount of a substance capable of modulating activity of LEMD3 in the vertebrate subject to thereby modulate TGFβ biological activity in the vertebrate subject.

LEMD3 antagonizes TGF-beta-driven Smad2/3 transcription in a stiffness-dependent fashion in both the nucleus and cytosol

Methods and compositions for modulating transforming growth factor-beta (TGFβ) biological activity in a vertebrate subject in need thereof. The methods involve administering to the vertebrate subject an effective amount of a substance capable of modulating activity of LEMD3 in the vertebrate subject to thereby modulate TGFβ biological activity in the vertebrate subject.

RNA interference agents for GST-PI gene modulation

This invention provides compounds, compositions and methods for modulating the expression of human GST-π using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. Provided are a range of siRNA structures, having one or more of nucleotides being modified or chemically-modified. Advantageous structures include siRNAs with 2′-deoxy nucleotides located in the seed region, as well as other nucleotide modifications.

RNA interference agents for GST-PI gene modulation

This invention provides compounds, compositions and methods for modulating the expression of human GST-π using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. Provided are a range of siRNA structures, having one or more of nucleotides being modified or chemically-modified. Advantageous structures include siRNAs with 2′-deoxy nucleotides located in the seed region, as well as other nucleotide modifications.

Compositions and methods for organ specific delivery of nucleic acids

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a SIN CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing SIN CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a SIN CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing SIN CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Long double-stranded RNA for RNA interference
11591600 · 2023-02-28 · ·

The present technology relates, in part, to long double-stranded RNA (dsRNA) (e.g., 30 or more base pairs) that inhibits gene expression.