Cel
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.
Dziedzina nauki
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- natural sciencesbiological sciencesbiochemistrybiomoleculeslipids
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- natural sciencesbiological sciencesgeneticsRNA
- engineering and technologynanotechnologynano-materials
Słowa kluczowe
Program(-y)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
System finansowania
HORIZON-ERC-POC - HORIZON ERC Proof of Concept GrantsInstytucja przyjmująca
80333 Muenchen
Niemcy