Custom iPSC Genome Editing

Create isogenic and reporter cell lines for disease modeling and drug discovery

Combining iPSC technology with precision genome editing 

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) system is the latest tool in genome editing. Harnessed from the adaptable immune mechanisms of bacteria, CRISPR/Cas has been modified to achieve efficient and specific alterations of genome sequence and gene expression in many species, including a wide variety of biomedically important cell types. By delivering the Cas protein and appropriate guide RNAs into a cell, the genome can be cut at any precise location allowing for specific gene disruption or replacement.

Cellular reprogramming and directed differentiation of iPS cells offer the possibility of generating many disease-relevant cell types from any genetic background. By combining Horizon Discovery's CRISPR genome editing with our iPS cell technology, we can create accurate cellular models of genetic diseases by introducing disease-causing mutations or correct mutations in the genomes of the disease-relevant cells. These models can help researchers better understand how specific genes contribute to disease pathogenesis at the molecular and cellular level in a tissue-specific nature. Furthermore, they represent valuable platforms for identifying drug targets and studying drug mechanisms of action, paving the way for developing new therapeutic interventions for many currently untreatable genetic diseases. 

What we can do for you

Construct and test CRISPR vectors - we discuss with you the vector design and targeting strategy

Expand your iPS cells as single cells under fully defined culture conditions

Sequence the targeted genome region

Design and synthesize the CRISPR guide RNA and donor DNA vector

Validate the custom guide RNA plasmid for nuclease efficiency

Electroporate the CRISPR guide RNA, Cas9 plasmids and the single stranded oligonucleotide into the iPS cell line

Clonal expansion of gene-edited iPS cell line

Screen targeted clones to verify correct genetic modification

Expand the verified iPS cell clones

Freeze down the verified iPS cell clones

Directed differentiation of iPS cells to multiple cell types, including cardiomyocytes, endothelial cells and neural cells. Find out more...

The turnaround time for the genome editing service is about 4 months. At least three frozen vials of donor cells per sample will be needed. One vial will be thawed and tested for mycoplasma. We will start iPS cell expansion once the quarantine is completed.

Talk to us about your project!



What our customers say...

"I've been able to grow all the cells that Axol generated for me from my patient lines. All the cells provided grew well and I'm very happy with the service as well as the quality of the cells."

 Dr David Millar, Human Molecular Genetics, Cardiff University

"Axol has provided us with more than a fast and reliable supply of functional cortical human iPSC derived neurons. It has also given us access to the collective expertise of Axol, whom we now view as much a collaborator as a company”

 Dr Eric Hill, Clinical and Systems Neuroscience, Aston University

"Axol Bioscience has been a valued and important supplier to our lab’s research since the company was founded. We began working with sample product and were rapidly convinced by the quality of the cells, such that we now have long term orders in place for 2 neural precursor lines. Additionally, we have recommended Axol cells to several of our closest collaborators. The cells supplied to us by Axol represent exceptional value for money, but, most importantly, save us a great deal of valuable research time. Throughout our dealings with the company, service and support have always been excellent.”

 Dr Paul Charlesworth, Physiology, University of Cambridge