Project description
A genetically-controlled breakthrough in RNA delivery
Human induced pluripotent stem cells (hIPSCs) hold immense potential for biomedical research. However, their genetic modification presents significant challenges. Existing delivery techniques lack precision and efficiency. In this context, the ERC-funded inteRNAlizer project will introduce a genetically controlled, cost-effective RNA delivery system, enabling precise gene expression and editing in diverse cell types, including hIPSCs and T cells. With high efficacy and biosafety comparable to S1 standards, inteRNAlizers stand as a promising alternative to conventional methods like lentiviruses. This innovation not only enhances preclinical research but also accelerates therapeutic cell system development, unlocking new avenues in gene delivery applications.
Objective
Human induced pluripotent stem cells (hIPSCs) have revolutionized the study of cell type-specific processes and the generation of organoids, tissues, and therapeutic cells for biomedical purposes.
However, the genetic modification of these cells, along with other difficult-to-transfect cells, poses a major challenge for performing high-throughput gene reporter and genetic perturbation assays and prevents us from fully exploiting the potential of hIPSCs.
Existing gene delivery techniques, such as lentiviruses or lipid nanoparticles, suffer from limitations in precision, biosafety, efficacy, and high production costs.
To overcome these limitations, our team has developed a novel approach called inteRNAlizers, which offers a genetically controlled cellular production process for non-viral RNA delivery systems.
inteRNAlizers can enable transient gene expression and modular gene editing in virtually any cell type, including differentiated hIPSC and T cells. The method demonstrates high efficacy and cost efficiency while maintaining biosafety levels comparable to S1 standards.
We aim to position inteRNAlizers as a promising alternative to lentiviruses and lipid nanoparticles, opening up new possibilities in gene delivery applications for preclinical research and therapeutic cell systems.
Fields of science
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- natural sciencesbiological sciencesbiochemistrybiomoleculeslipids
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- natural sciencesbiological sciencesgeneticsRNA
- engineering and technologynanotechnologynano-materials
Keywords
Programme(s)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Funding Scheme
HORIZON-ERC-POC - HORIZON ERC Proof of Concept GrantsHost institution
80333 Muenchen
Germany