iPSC-derived cell products
Hepatocyte-like cells and Intestinal organoids available
DefiniGEN offer a unique induced Pluripotent Stem Cells (iPSC) derived disease modeling platform to support pre-clinical research into liver and metabolic disease models.
Utilizing our extensive experience in differentiation, we offer a suite of disease modeling services and bioassay endpoints to help profile compounds and gene therapies for the evaluation of delivery and efficacy of therapeutic candidates.
Our in-house service offering provides expertise from our seasoned team of scientists who can help optimize experiments enabling the accelerated generation of disease modeling data. We also offer several cryopreserved cell vials that can be utilized for your own internal research or evaluation.
The cryopreserved cells exhibit good recovery after thawing, maintain morphology, display key functionality markers, and offer longer assay windows than primary human cells.
Explore our Hepatocyte Models
iPSC-derived Human Hepatocytes: Alpha-1 Antitrypsin Deficiency
DefiniGEN provides iPSC-derived Alpha-1 Antitrypsin deficiency (A1ATD) hepatocytes which are highly functional and showcase a strong diseased phenotype in vitro.
Cells are derived from either A1ATD patients or generated through CRISPR engineering and carry the 'Z' (E342K) point mutation in the SERPINA1 gene. This mutation leads to the accumulation of polymeric aggregates of the proteins that ultimately cause liver and lung damage. DefiniGEN's iPSC-derived A1ATD hepatocytes represent an optimized disease model for drug discovery applications and are an effective tool for elucidating the underlying mechanisms of the disease.
DefiniGEN offer homozygous patient-derived A1AT cells and both homozygous and heterozygous CRISPR-engineered A1ATD lines.
iPSC-derived Human Hepatocytes: Wilson's Disease
DefiniGEN's iPSC-derived Wilson's Disease (WD) hepatocytes provide a relevant disease modeling platform to study this rare disease.
Cells are generated through CRISPR engineering and carry the homozygous H1069Q mutation in the ATP7B gene. This mutation causes a decrease in the protein function and leads to excessive accumulation of copper that can cause damage to the liver, brain, and other vital organs. DefiniGEN's iPSC-dervied WD hepatocytes are a powerful tool for drug discovery applications and can help investigate this monogenic disease and other conditions related to oxidative stress.
iPSC-derived Human Hepatocytes: Glycogen Storage Disease Type 1 Alpha
DefiniGEN provides iPSC-derived Glycogen Storage Disease Type 1 Alpha (GSD1A) hepatocytes that are suitable to study disorders related to glycogen storage. This genetic disease results from a deficiency in the glucose-6 phosphatase (G6P) enzyme which impairs the ability of the liver to produce free glucose from glycogen.
These cells can offer disease modeling and drug discovery researchers a tool to develop new therapies for this and similar lysosomal storage diseases or to further the understanding of the disease mechanisms.
We offer two patient lines with different genotypes: homozygous R83C in G6PC and compound heterozygous with G222R and Q347X mutations in G6PC.
iPSC-derived Human Hepatocytes: Non-Alcoholic Fatty Liver Disease
Non-alcoholic fatty liver disease (NAFLD) is characterized by the accumulation of fat within the liver that can lead to inflammation, fibrosis, and hepatocellular carcinoma. Although it's not a monogenic disease, several variants have been associated with increased risk to develop NAFLD.
DefiniGEN's iPSC-derived NAFLD hepatocytes have been edited to include some of the most common genetic variants implicated in the disease. One of the lines includes the missense homozygous mutation I148M in the gene coding for Patatin-like phospholipase domain-containing protein 3 (PNPLA3) and another line harbors the homozgyous E167K mutation in the gene encoding for Transmembrane 6 Superfamily Member 2 (TM6SF2).
DefiniGEN's iPSC-derived NAFLD hepatocytes are an important tool for disease modeling and drug discovery for this common liver disease.
iPSC-derived Human Intestinal Organoids: Wild-Type
DefiniGEN's iPSC-derived Wild-Type (WT) intestinal organoids provide a unique in vitro system to model the human intestine. The organoids display a polarized epithelium and harbor a mixture of cell types normally present in the primary intestinal epithelial barrier in vivo, including goblet cells, Paneth cells, enterocytes, and enteroendocrine cells. The cells can be used for drug absorption, metabolism, and the modeling of infectious disease.
DefiniGEN's intestinal monolayer constitutes an effective application to model the permeability of the human intestine and investigate drug absorption and metabolism. Following a simple and standardized protocol, cells from DefiniGEN's intestinal organoids can be grown as a monolayer in a similar manner to standard Caco-2 immortalized cell lines with the key benefit of greatly increased biological relevance, as the cells have primary human intestinal cell function, metabolism and genetics.