Descrizione del progetto
Una svolta geneticamente controllata nella somministrazione di RNA
Le cellule staminali umane pluripotenti indotte (hIPSC) hanno un immenso potenziale per la ricerca biomedica. Tuttavia, la loro modifica genetica presenta sfide significative. Le tecniche di somministrazione esistenti mancano di precisione ed efficienza. In questo contesto, il progetto inteRNAlizer, finanziato dal CER, introdurrà un sistema di rilascio di RNA geneticamente controllato ed economicamente vantaggioso, che consentirà l’espressione e l’editing genico preciso in diversi tipi di cellule, comprese le hIPSC e le cellule T. Con un’elevata efficacia e una biosicurezza paragonabile agli standard S1, inteRNAlizer rappresenta una promettente alternativa ai metodi convenzionali come i lentivirus. Questa innovazione non solo migliora la ricerca preclinica, ma accelera anche lo sviluppo di sistemi cellulari terapeutici, aprendo nuove strade alle applicazioni di somministrazione genica.
Obiettivo
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.
Campo scientifico
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- natural sciencesbiological sciencesbiochemistrybiomoleculeslipids
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- natural sciencesbiological sciencesgeneticsRNA
- engineering and technologynanotechnologynano-materials
Parole chiave
Programma(i)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Argomento(i)
Meccanismo di finanziamento
HORIZON-ERC-POC - HORIZON ERC Proof of Concept GrantsIstituzione ospitante
80333 Muenchen
Germania