Project description
Next-gen gene therapy with barcoded lipid nanoparticles
Gene therapy could be used to treat a range of diseases, but current viral methods face limitations, such as immune reactions, size restrictions and complex production. While synthetic lipid nanoparticles (LNPs) offer a scalable and cost-effective alternative, efficient gene transfer is a challenge. The ERC-funded AGTC project will develop barcoded LNPs that insert genes directly into T cells in vivo, allowing researchers to track delivery, optimise design and study biological outcomes. Focusing on nanomedicine design, precise targeting and T cell biology, AGTC aims to unlock the principles behind effective DNA delivery. The project promises to advance synthetic gene therapies.
Objective
Innovation: We recently developed a targeted lipid nanopar We recently developed a targeted lipid nanoparticle (tLNP) that can insert a gene directly into the genome of T cells in vivo, with efficiency comparable to viral methods. This innovation creates a unique opportunity to track gene delivery using barcoded synthetic vectors. These barcodes help us understand the key factors needed for effective DNA delivery and the biological consequences of gene transfer.
Background: Gene therapy holds great promise for treating various diseases. Viral vectors are currently the most common method for gene transfer, but they come with limitations like size restrictions on nucleic acids, triggering immune responses, and complicated production processes. Synthetic vectors like LNP have made significant strides, as shown by the success of mRNA COVID-19 vaccines, which demonstrate the efficacy, scalability and cost-effectiveness of LNP. However, LNP have struggled with efficient gene transfer. Improving this is challenging due to the vast nanomedicine design space and poor correlation between in vitro and in vivo results. Barcoding technology has revolutionized LNP design by allowing higher throughput screening, enabling comprehensive sampling of the design space in living organisms. To apply this to gene transfer, a working prototype is necessary. Our recent success with LNP-mediated gene transfer serves as this foundation for rational optimization of synthetic vectors using DNA-encoded peptide barcodes.
Objectives: The AGTC project will explore three main research lines:
1. Nanomedicine Design: Efficiently explore the design space to optimize LNP.
2. Targeting: Investigate precision and flexibility of targeting, particularly in T cells.
3. T Cell Biology: Link gene transfer to biological activity, focusing on CAR T cells.
Outcomes: AGTC will enhance our understanding of what drives effective DNA delivery, paving the way for a new generation of synthetic gene transfer therapies.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- natural sciences biological sciences genetics DNA
- natural sciences biological sciences biochemistry biomolecules lipids
- medical and health sciences medical biotechnology nanomedicine
- medical and health sciences basic medicine pharmacology and pharmacy pharmaceutical drugs vaccines
- engineering and technology nanotechnology nano-materials
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.1.1 - European Research Council (ERC)
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Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
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Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC - HORIZON ERC Grants
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Call for proposal
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Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2024-ADG
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3584 CX Utrecht
Netherlands
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