Nucleic Acids for Future Gene Editing, Immunotherapy and Epigenetic Sequence Modification

Nucleic acid (NA) therapeutics are expected to yield major advances in the treatment of human diseases, especially cancer. As NAs behave in a fundamentally different way than the small molecules traditionally forming the backbone of drug formulation, recent advances, such as gene editing, cancer immunotherapy, and epigenetic base manipulation overcome the limitations otherwise inherent to NAs.

This new and fast growing field underpins Europe’s biotechnology and pharmaceutical industries and requires highly qualified experts in nucleic acid chemistry, biomaterials development and chemical biology.

NATURE-ETN will leverage recent breakthrough discoveries in epigenetic manipulation, gene editing, small molecule DNA targeting, and rapid gene/transgene detection to extend the boundaries of molecular medicine and provide new tools for treating cancer and monogenetic diseases. The ESRs trained within NATURE-ETN will acquire the necessary knowledge and skillset to fill high-functioning industry jobs, but also to maintain and advance Europe’s competitiveness and innovation capacity.

The scientific work is divided into three Work Packages (WP):

  • WP1 Extending the boundaries of gene editing technologies: CRISPR sgRNA strands will be engineered for light-controlled activation and live-cell imaging. A new route to gene editing using TFO hybrids will be pioneered and studied by X-ray crystallography.
  • WP2 Developing therapeutic oligonucleotides for cancer immunotherapy: Manipulation of T-cells for CAR-T immunotherapy will be undertaken with cGAMP and therapeutic mRNAs; recent discoveries with engineered polymerases will be applied.
  • WP3 Sequencing and imaging of epigenetic bases: New methods for epigenetic base oxidation will be applied to mainstream sequencing technologies.