Abstract: The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.

Abstract: Compositions and methods for inserting a nucleic acid encoding a product of interest into a genomic safe harbor locus in a cell, a population of cells, or a subject or for expressing a nucleic acid encoding a product of interest from a genomic safe harbor locus in a cell, a population of cells, or a subject are provided. Also provided are cells or populations of cells comprising a nucleic acid construct comprising a coding sequence for a product of interest inserted into a genomic safe harbor locus. Also provided are methods of identifying genomic safe harbor loci for use in specific cell or tissue types.

Abstract: The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.

Abstract: The present invention relates to a method of determining the risk of drug induced arrhythmia using stem cell derived cardiomyocytes in a high-throughput impedance or multi-electrode array assay.

Abstract: The invention relates to the compounds of formula I and pharmaceutically acceptable salts and esters thereof, wherein R1-R11 are as defined in the description and claims. In addition, the present invention relates to methods of manufacturing and using the compounds of formula I as well as pharmaceutical compositions containing such compounds. The compounds of formula I are useful in differentiating stem cells into more mature or adult-like hepatocytes for use as drug screening platforms and in disease modeling applications.

Abstract: The invention relates to the compounds of formula I and pharmaceutically acceptable salts and esters thereof, wherein R1-R11 are as defined in the description and claims. In addition, the present invention relates to methods of manufacturing and using the compounds of formula I as well as pharmaceutical compositions containing such compounds. The compounds of formula I are useful in differentiating stem cells into more mature or adult-like hepatocytes for use as drug screening platforms and in disease modeling applications.

Abstract: The invention relates to the compounds of formula I and pharmaceutically acceptable salts and esters thereof, wherein R1-R11 are as defined in the description and claims. In addition, the present invention relates to methods of manufacturing and using the compounds of formula I as well as pharmaceutical compositions containing such compounds. The compounds of formula I are useful in differentiating stem cells into more mature or adult-like hepatocytes for use as drug screening platforms and in disease modeling applications.

Abstract: The invention relates to the compounds of formula I and pharmaceutically acceptable salts and esters thereof, wherein R1-R11 are as defined in the description and claims. In addition, the present invention relates to methods of manufacturing and using the compounds of formula I as well as pharmaceutical compositions containing such compounds. The compounds of formula I are useful in differentiating stem cells into more mature or adult-like hepatocytes for use as drug screening platforms and in disease modeling applications.

Abstract: The present invention relates to a method of determining the risk of drug induced teratogenicity using pluripotent stem cells.

Abstract: The present invention relates to a method of determining the risk of drug induced arrhythmia using stem cell derived cardiomyocytes in a high-throughput impedance or multi-electrode array assay.