C12N2310/14

OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN MODULATION OF SMALL NON-CODING RNAs

Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.

Dually Derivatized Chitosan Nanoparticles and Methods of Making and Using the Same for Gene Transfer In Vivo
20180008720 · 2018-01-11 ·

Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.

DOUBLE-STRANDED OLIGONUCLEOTIDE MOLECULES TO DDIT4 AND METHODS OF USE THEREOF

Provided herein are double stranded nucleic acid molecules, compositions comprising same and methods of use thereof for the treatment of a subject wherein expression of DDIT4 is associated with the etiology or progression of a disease or disorder in the subject. The compounds are preferably chemically synthesized and modified dsRNA molecules.

COMPOSITIONS AND METHODS TO PROMOTE ERYTHROPOIESIS
20180010129 · 2018-01-11 ·

Described herein are compositions and methods for enhancing erythropoiesis in an individual in need thereof. Specifically agents that decrease the expression of Exosc10, such as inhibitory nucleic acid molecules, produce an increase in red blood cell production in the individual.

INHIBITION OF PRMT5 TO TREAT MTAP-DEFICIENCY-RELATED DISEASES

The invention provides novel personalized therapies, kits, transmittable forms of information and methods for use in treating patients having cancer, wherein the cancer is MTAP-deficient and/or MTA-accumulating and thus amenable to therapeutic treatment with a PRMT5 inhibitor. Kits, methods of screening for candidate PRMT5 inhibitors, and associated methods of treatment are also provided.

Double-stranded ribonucleic acid as control against insects

A composition for use in formulations for controlling insect populations, including populations of mosquito and flies. The composition comprises one or more double-stranded constructs inhibitory to RNA transcription of ribosomal proteins. The invention also relates to method of using the compositions in formulations to inhibit insect populations.

COMPOSITIONS AND METHODS FOR MONITORING, DIAGNOSIS, PROGNOSIS, DETECTION, AND TREATMENT OF CANCER
20180010195 · 2018-01-11 ·

The present invention relates to the field of cancer and inflammation, and in particular methods of monitoring, diagnosis, prognosis, detecting, treating and preventing cancer and inflammation conditions. The use of composition comprises method of monitoring, diagnosis, prognosis, and detecting cancer and inflammation with EN2 and/or SATB2 expression and/or activity. The pharmaceutical composition will further comprise agents that inhibit EN2 and/or SATB2 expression or activity.

METHOD OF PRODUCING EXOSOMES FROM STEM CELLS AND USE THEREOF
20180010133 · 2018-01-11 ·

A method of producing induced exosomes, the method comprising: contacting an isolated population of stem cells with an amount of a prostaglandin E receptor 4 (EP4) antagonist effective for inducing release of exosomes, whereby induced exosomes are released from the stem cells, and isolating the induced exosomes.

RNA interference delivery formulation and methods for malignant tumors

This invention provides formulations for use in distributing RNAi molecules targeted to a human GST-π for treating a malignant tumor in a subject. The formulation can include nanoparticles composed of an ionizable lipid, a DSPE lipid, and additional lipids. A drug product can be made by lyophilization of the formulation. This invention further provides methods for ameliorating or treating a malignant tumor by administering a therapeutically effective amount of a formulation containing the RNAi agents.

THERAPEUTIC TARGETING OF SET1B/COMPASS PATHWAY FOR TREATING CANCERS
20180009870 · 2018-01-11 · ·

Disclosed are methods for treating Set1/COMPASS-associated cancers characterized by expression of Set1B/COMPASS. The methods typically include administering a therapeutic amount of an inhibitor of the Set1B/COMPASS pathway and/or an agonist for a target that is negatively regulated by the Set1B/COMPASS pathway.