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Efficient, safe, and cost-efficient RNA delivery vehicles for hard-to-transfect pre-clinical and therapeutic cells.

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 (EuroSciVoc)

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Topic(s)

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Funding Scheme

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HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

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(opens in new window) ERC-2023-POC

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Host institution

TECHNISCHE UNIVERSITAET MUENCHEN
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 150 000,00
Address
Arcisstrasse 21
80333 Muenchen
Germany

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Region
Bayern Oberbayern München, Kreisfreie Stadt
Activity type
Higher or Secondary Education Establishments
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Total cost

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Beneficiaries (1)

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