Optimized human cell products for research
and drug discovery

Custom Disease Model – CRISPR Gene-edited

The OptiDIFF technology was developed from world-leading research undertaken at the University of Cambridge and has been demonstrated on a range of cell types including liver, pancreatic and lung cell types. This platform has been combined with the market-leading CRISPR gene-editing technology provided by Horizon Discovery Ltd, to offer a unique resource for custom cell line development. This enables the precise introduction of any desired mutation and the rapid production of optimized disease models of monogenic and complex diseases including Diabetes and NAFLD. Ultimately the client receives fully differentiated, rigorously QCed cell products which are genotype and phenotype verified in a highly relevant physiological background.

 

Definigen OptiDIFF Platform

Horizon Discovery Ltd

6 step process for the generation of custom liver and pancreatic disease models powered by Horizon Discovery CRISPR gene-editing technology

Implementation

Generation of custom liver and pancreatic disease models

Timeline

Custom Disease Model – CRISPR Gene-Edited projects are typically completed within 4-6 months. Actual timelines will vary depending on the unique aspects of each bespoke project.

Selected platform publications

Generation of Hepatocytes from Pluripotent Stem Cells for Drug Screening and Developmental Modeling.  Gieseck RL 3rd, Vallier L, Hannan NR. Methods Mol Biol. 2015;1250:123-42.

Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation. Sampaziotis F, Vallier L et al Nature Biotechnol. 2015 Aug; 33(8): 845–852.

Generation of Distal Airway Epithelium from Multipotent Human Foregut Stem Cells. Hannan NR, Sampaziotis F, Segeritz CP, Hanley NA, Vallier L. Stem Cells Dev. 2015 Jul 15;24(14):1680-90.

Production of hepatocyte-like cells from human pluripotent stem cells. Hannan NR, Vallier L et al. Nature Protocols. 2013 Feb;8(2):430-7.

Inhibition of activin/nodal signalling is necessary for pancreatic differentiation of human pluripotent stem cells. Cho C , Hannan NR, Vallier L et al. Diabetologia. 2012 Dec 55(12):3284-95.

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Yusa K, Rashid ST, Vallier L et al. Nature. 2011 Oct 12;478(7369):391-4.

Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells. Rashid ST, Lomas DA, Vallier L et al. J Clin Invest. 2010 Sep;120(9):3127-36.

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