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.

A method for screening an active ingredient of a saltiness enhancer, the screening method including the following steps: (i) a step for determining whether a test substance is a compound capable of promoting functional expression of the TMC4 gene or TMC4 protein; and (ii) a step for selecting, as an active ingredient of a saltiness enhancer, a test substance that has been determined in step (i) to be a compound capable of promoting functional expression of the TMC4 gene or TMC4 protein.

The disclosure provides a composition for suppressing inflammation comprising at least one substance that disrupts a stem-loop structure in the 3′ untranslated region of a Regnase-1 mRNA, wherein the stem-loop structure is at least one stem-loop structure selected from a first stem-loop structure formed in a region corresponding to positions 231 to 245 of SEQ ID NO: 1 and a second stem-loop structure formed in a region corresponding to positions 424 to 442 of SEQ ID NO: 1.

Based on the discovery that MBLAC1 is a specific, high-affinity target for Ceftriaxone (Cef), MBLAC1 may be used for identifying treatments for addiction to substances of abuse. Methods for identifying therapeutic agents for treatment of addiction to a substance of abuse include using an assay to determine if a test agent is capable of binding to MBLAC1 or disrupting binding between MBLAC1 protein and Cef, and identifying such a test agent as a candidate therapeutic agent for treatment of addiction to a substance of abuse. MBLAC knock-out (KO) animals, methods of use thereof, and kits are used for identifying a therapeutic agent that reduces the actions of at least one substance of abuse. Methods also include using cellular extracts from tissue or cultured cells taken from wild-type (WT) MBLAC1 and MBLAC1 KO animals for screening for novel, Cef-like molecules in vitro, and using cells from a MBLAC1 KO animal to test for Cef-like actions of a test molecule.

Provided herein are methods for determining a rate of DNA double strand repair on a DNA strand in a cell that include (a) delivering a reporter gene, a gene-editing agent, and a gene-repair template into a cell, wherein the gene-editing agent generates a DNA double strand break on the DNA strand; (b) detecting a change in reporter gene expression, wherein the change in reporter gene expression indicates the presence of a DNA double strand repair event; and (c) analyzing the change in reporter gene expression, thereby determining the rate of DNA double strand repair on the DNA strand in the cell.

The present invention relates to a new cell based assay for combined determination of antibody or ligand binding and function in the same vial.

The present invention relates to microfluidic fluidic devices, methods and systems as microfluidic kidney on-chips, e.g. human Proximal Tubule-Chip.

The present disclosure relates to compositions and methods for the preparation and use of free fatty acids as crystals and in solution, optionally in array format, for assay of lipotoxicity and related effects (in certain cases, indicators of diseases or disorders, such as type II diabetes) in contacted cells. Identification and therapeutic targeting of high-value gene targets discovered via joint assessment of lipotoxicity/transcriptome data and genetic association study data are also provided.

Disclosed is a spheroid liver organoid comprising hepatic lineage cells such as human hepatocytes, hepatic stellate cells, and liver sinusoidal endothelial cells. Also provided are methods of using spheroid liver organoids for applications related to drug screening and toxicity screening. In particular, spheroid liver organoids are useful for high-throughput screens to identify compounds having efficacy for treating liver disease.

The present disclosure provides an in vitro cell based potency assay to determine the relative potency of a composition, including a pharmaceutical composition, comprising an mRNA encapsulated in a lipid nanoparticle (LNP) as compared to a reference sample. Also provided is a process for releasing or accepting a batch of a pharmaceutical composition comprising an mRNA encapsulated in an LNP using the in vitro cell based potency assay. The methods and processes described comprise (i) transfecting a population of cells with a test sample of the composition, (ii) transfecting a different population of cells with a reference sample of the pharmaceutical composition, wherein the cells in step (ii) are the same cell type as the cells in step (i); (iii) detecting the amount of expression of a polypeptide encoded by the mRNA in the transfected cells; and comparing the amount of expression, thereby determining the relative in vitro potency of the composition.