The GetRadi consortium established several cellular models for the rare diseases. These disease models will be corrected with different gene editing technologies including CRISPR gene editing and novel variants of it, which are less prone to adverse effects. In addition, novel regulators of the gene editing process have been identified that might be targets for increasing repair efficiency.
To introduce the gene editing tools into the target cells, GetRadi is testing and optimising different methodologies including electroporation, lipid nanoparticles, exosomes and adenoviral particles.
To increase the safety of gene therapy, variants of Cas9 with a reduced risk for immune reaction are investigated. Moreover, GetRadi is trying to perform gene editing of stem cells of rare disease patient by automated cell culturing equipment, in order optimize and standardize the therapy in compliance with good manufacturing practice.
GetRadi furthermore developed reporter cell lines and mice for testing of the efficiency and safety of in vivo and ex vivo gene therapy techniques, which includes gene editing tools, transfer of gene editing tools to target cells, sustained treatment success, and immune reactions.
Using the newly established tools and optimized methodologies described above, research of the GetRadi consortium will contribute to novel, more efficient, more safe, and less expensive therapy or rare diseases.
GetRadi aims to publish results from research on rare diseases in peer-reviewed articles. Seven review articles and one scientific article have so far been published, and more are to follow within the remaining project period.
