PASCAL-MID is a newly established CRISPR/Cas9 research center founded on empirical experience with the technique. The background for the generation of thi 'brickless' center is that current treatment options for rare monogenic disorders are expensive and have low success rates. CRISPR/Cas9 enables a new type of genetic treatment, and with a large research grant from the Innovation Fund Denmark we will ensure that Aarhus University and Aarhus University Hospital can perform world-leading, cutting edge research in this new technology.

The purpose of the center is to develop CRISPR/Cas-based treatments for monogenic disorders with a focus on primary immunodeficiencies. The aim is to establish the necessary research infrastructure to be able to offer CRISPR/Cas-based gene therapies as standard treatment option. This infrastructure will be the foundation for future development of gene therapies in Denmark. Success criteria include functional validation of gene editing in primary cells and validation of standard operating procedures (SOPs) for approval of clinical trials. The synergism established between researchers and clinicians will secure a fully functional facility for correction of genetic defects in hematopoietic stem cells. 


With the discovery and development of the CRISPR/Cas technology in recent years, there is now the possibility as well as a clear and present need for carrying CRISPR/Cas to the clinic to enable the use of the patient cells for auto-transplantation after gene correction. The CRISPR/Cas stem cell gene editing facility that we aim to establish will include a comprehensive program encompassing all aspects of this technology, ranging ultimately from the most basic aspects of gene editing efficiency and safety to clinical application. This will allow for a project that has the potential to generate ground-breaking scientific results, major publications in international high-impact journals, patenting, spin-out companies, and not least novel treatment modalities aiming at a cure for patients with severe PIDs and other monogenic diseases with potential great value and benefit society.