Genome editing

Editing for basic and translational research

Human-induced pluripotent stem cells (hiPSCs) when combined with gene editing methods including CRISPR/Cas9 gene present two instruments of basic and translational research, which both allow to acquire deep insight about the molecular bases of many diseases but also to develop pharmacological research.

Genetically edited iPSC-derived models are used to better understand how specific genes contribute to disease pathogenesis at the molecular and cellular level in a tissue-specific nature.

These models they represent valuable platforms for identifying drug targets and studying drug mechanisms of action, paving the way for developing new therapeutic interventions for many genetic diseases that are currently untreatable.

Our gene-editing offering

Our custom cellular reprograming service and directed differentiation of iPSCs offers the possibility of generating many disease models from any genetic background.

By combining the power of CRISPR-Cas9 genome editing with our iPSC technology, we can create accurate cellular models of genetic conditions by introducing disease-causing mutations into healthy control cell lines.
We can also correct those mutations in genomes of patient-derived cells to provide the matched control cell line.

Learn more

• De Masi, C., Spitalieri, P., Murdocca, M. et al. Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery. Hum Genomics 14, 25 (2020).