Efficient, safe, and cost-efficient RNA delivery vehicles for hard-to-transfect pre-clinical and therapeutic cells.
The project aims to develop inteRNAlizers, a novel non-viral RNA delivery system for efficient and safe gene modification in hIPSCs and other cells, enhancing gene delivery for research and therapies.
Projectdetails
Introduction
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.
Limitations of Existing Techniques
Existing gene delivery techniques, such as lentiviruses or lipid nanoparticles, suffer from limitations in:
- Precision
- Biosafety
- Efficacy
- High production costs
Novel Approach: inteRNAlizers
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.
Features of inteRNAlizers
- Enables transient gene expression
- Supports modular gene editing in virtually any cell type, including differentiated hIPSCs and T cells
- Demonstrates high efficacy and cost efficiency
- Maintains biosafety levels comparable to S1 standards
Future Directions
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.
Financiële details & Tijdlijn
Financiële details
Subsidiebedrag | € 150.000 |
Totale projectbegroting | € 150.000 |
Tijdlijn
Startdatum | 1-12-2023 |
Einddatum | 31-5-2025 |
Subsidiejaar | 2023 |
Partners & Locaties
Projectpartners
- TECHNISCHE UNIVERSITAET MUENCHENpenvoerder
Land(en)
Vergelijkbare projecten binnen European Research Council
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In Vivo CRISPR-Based Nanoplatform for Gene Editing: A New Disruptive Avenue for Non-Invasive Treatment of Genetic Brain DiseasesThis project aims to develop a novel nanoplatform for the safe and efficient delivery of CRISPR gene editing technology to treat genetic brain diseases non-invasively. | ERC Consolid... | € 2.249.895 | 2022 | Details |
Kits for advanced polymer-lipid nanocarriers for targeted delivery of RNAs to cardiac and skeletal muscle cellsPOLIRNA aims to develop a versatile platform for safe and efficient RNA delivery to target multiple cell types, enhancing preclinical research in cardiac and muscle-related diseases. | ERC Proof of... | € 150.000 | 2023 | Details |
Transcriptional Engineering of Hematopoietic Stem Cells using CRISPRThis project aims to enhance hematopoietic stem cell therapies by using repurposed CRISPR/Cas systems for precise transcriptional manipulation of key genetic pathways. | ERC Starting... | € 1.499.923 | 2022 | Details |
Dissecting the molecular regulation of hematopoietic stem cell emergence using pluripotent stem cells to improve ex vivo therapies
This project aims to develop methods for generating and expanding hematopoietic stem cells from patient-specific induced pluripotent stem cells to overcome transplantation barriers and enhance therapies.
Scaling up iPSC Expansion and Differentiation using Recombinant Bioemulsions
The project aims to develop scalable, regulatory-compliant bioemulsions using engineered protein nanosheets for efficient iPSC culture and differentiation, enhancing cell manufacturing and biotech market growth.
In Vivo CRISPR-Based Nanoplatform for Gene Editing: A New Disruptive Avenue for Non-Invasive Treatment of Genetic Brain Diseases
This project aims to develop a novel nanoplatform for the safe and efficient delivery of CRISPR gene editing technology to treat genetic brain diseases non-invasively.
Kits for advanced polymer-lipid nanocarriers for targeted delivery of RNAs to cardiac and skeletal muscle cells
POLIRNA aims to develop a versatile platform for safe and efficient RNA delivery to target multiple cell types, enhancing preclinical research in cardiac and muscle-related diseases.
Transcriptional Engineering of Hematopoietic Stem Cells using CRISPR
This project aims to enhance hematopoietic stem cell therapies by using repurposed CRISPR/Cas systems for precise transcriptional manipulation of key genetic pathways.
Vergelijkbare projecten uit andere regelingen
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NOn-VIral gene modified STEM cell therapyThis project aims to develop a high-throughput protocol for producing gene-corrected CAR T cells and blood stem cells using optimized photoporation and CRISPR technology for enhanced clinical application. | EIC Pathfinder | € 3.644.418 | 2022 | Details |
New Prime Editing and non-viral delivery strategies for Gene TherapyThis project aims to develop non-viral delivery systems and novel prime editors to enhance gene editing efficiency and safety for treating Sickle Cell Disease and other genetic disorders. | EIC Pathfinder | € 4.406.097 | 2022 | Details |
AI-powered platform for autologous iPSC manufacturingThe project aims to develop an AI-guided microfluidic device for the standardized, cost-effective mass production of personalized iPSCs to enhance cancer therapies and tissue regeneration. | EIC Pathfinder | € 3.999.225 | 2022 | Details |
Intelligent design of adenovirus vectors (iAds)The project aims to develop innovative, engineered adenovirus vectors for targeted gene therapy in heart and brain diseases by leveraging multi-national expertise and advanced design techniques. | EIC Pathfinder | € 3.443.137 | 2023 | Details |
Exploiting ex vivo expansion and deep multiomics profiling to bring novel, efficient and safer hematopoietic stem cell gene therapies to clinical applicationThis project aims to innovate hematopoietic stem cell identification and engineering through advanced culture techniques and multiomics profiling, enhancing gene therapy for blood disorders and cancer. | EIC Pathfinder | € 3.797.562 | 2022 | Details |
NOn-VIral gene modified STEM cell therapy
This project aims to develop a high-throughput protocol for producing gene-corrected CAR T cells and blood stem cells using optimized photoporation and CRISPR technology for enhanced clinical application.
New Prime Editing and non-viral delivery strategies for Gene Therapy
This project aims to develop non-viral delivery systems and novel prime editors to enhance gene editing efficiency and safety for treating Sickle Cell Disease and other genetic disorders.
AI-powered platform for autologous iPSC manufacturing
The project aims to develop an AI-guided microfluidic device for the standardized, cost-effective mass production of personalized iPSCs to enhance cancer therapies and tissue regeneration.
Intelligent design of adenovirus vectors (iAds)
The project aims to develop innovative, engineered adenovirus vectors for targeted gene therapy in heart and brain diseases by leveraging multi-national expertise and advanced design techniques.
Exploiting ex vivo expansion and deep multiomics profiling to bring novel, efficient and safer hematopoietic stem cell gene therapies to clinical application
This project aims to innovate hematopoietic stem cell identification and engineering through advanced culture techniques and multiomics profiling, enhancing gene therapy for blood disorders and cancer.