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Minimal Systems for Spatiotemporal Control of Gene Expression as Self-manufacturing Gene Therapeutics

Project description

Improving gene therapy delivery through self-regulating systems

The safe and precise delivery of gene therapies is necessary to ensure widespread clinical application. Backed by the Marie Skłodowska-Curie Actions programme, the Gene-Self-Expression project is designing a system that imitates the way living cells control gene expression in both space and time. By working with lipid-membrane compartments as tunable environments, researchers aim to develop a platform that can produce therapeutic mRNA when and where it is needed. Their goal is to create delivery systems that manufacture themselves, potentially revolutionising the development and application of gene therapies. This effort brings together expertise from chemistry, biology, physics, and engineering.

Objective

The development of effective gene therapies is hindered by limitations in current delivery methods, such as the use of lipid nanoparticles (LNPs) and extracellular vesicles (EVs). We propose a novel approach to overcome these limitations by constructing a self-organizing minimal system capable of producing therapeutic mRNA. Our system leverages the power of lipid membrane compartments to control and regulate gene expression, mimicking the sophisticated spatiotemporal control found in living cells.

Our research will focus on three main objectives: (1) investigating the activity of transcriptional and translational machinery in the proximity of lipid membranes, (2) designing and constructing a minimal system that exhibits spatiotemporal self-organization of gene expression, and (3) developing self-manufacturing platforms for the delivery and development of gene therapeutics.

By combining expertise in chemistry, biology, physics, and engineering, we aim to create a highly reproducible and efficient system for producing therapeutic mRNA. This will pave the way for the development of safer, more targeted, and more effective gene therapies, ultimately revolutionizing the treatment of various diseases.

Fields of science (EuroSciVoc)

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Keywords

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Programme(s)

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Topic(s)

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Funding Scheme

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HORIZON-TMA-MSCA-PF-EF - HORIZON TMA MSCA Postdoctoral Fellowships - European Fellowships

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Call for proposal

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(opens in new window) HORIZON-MSCA-2024-PF-01

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Coordinator

MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN EV
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.

€ 217 965,12
Address
HOFGARTENSTRASSE 8
80539 MUNCHEN
Germany

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Region
Bayern Oberbayern München, Kreisfreie Stadt
Activity type
Research Organisations
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Total cost

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