Designer recombinases platform for precise genome surgery
GO-Bio round 8
Prof. Frank Buchholz
Faculty of Medicine, Carl Gustav Carus UCC/Medical Systems Biology
Beneficiary: Technical University of Dresden
Genome editing has revolutionised the life sciences more than any other technology in recent years: the term refers to different molecular-biological tools that enable an organism's genetic code to be altered in a targeted manner. It not only opens up brand new doors for research itself, but also for medicine – genome-editing tools can also contribute to curing genetic diseases through 'molecular repair'.
The CRISPR/Cas system is currently the best-known tool for genome surgery. It is efficient and easy to use. It also has some drawbacks in terms of clinical application: the gene scissors cause incorrect edits to the genome known as 'off-target effects', undesirable when it comes to human medicinal use. Disputes over patents have also made it difficult to translate the technology into medical use. At the same time, many billions of US dollars have been invested in setting up new genome-editing companies lately.
The Dresden-based team led by Frank Buchholz has developed a whole new kind of genome-editing technology that is superior to the current procedure in many ways: it is based on sequence-specific recombination (SSR). After years of research, the team has been able to perfect the enzyme to allow it to edit cell genomes with great precision and use them extremely safely. The biotechnologists combined their own software with molecular evolution to tailor their recombinase enzymes to a particular target area. Designer recombinases can address almost all genetic diseases.
The team will build on the designer recombinases in a project entitled "RecTech" by establishing a safe and extremely precise market platform for genome surgery. It will target patent-protected curative therapies for genetic diseases in particular. The first GO-Bio phase aims to reduce the requisite development period for the manufacture of new designer recombinases. A previously developed recombinase will also see clinical development. The team plans on establishing an innovative genome-editing start-up that can grow into the leading manufacturer of recombinases for therapeutic applications.