Project description
Advancing precision tools for genome editing
Genome editing is opening new possibilities for treating genetic diseases via the correction of harmful mutations in the genome. However, many DNA-editing tools have raised safety concerns as they can cause off-target effects. To address this, the ERC-funded DC-PGE project is pioneering a fresh approach. Instead of relying on conventional enzymes that require host cell repair, researchers propose to engineer enzymes whose activity can be tightly controlled to modify only the intended DNA sequences. The project’s aim is to establish a versatile editing platform that can be adapted to target almost any gene. The approach will be validated for the treatment of severe genetic disorders, and if successful, it could transform genome editing tools.
Objective
Genome editing in human therapies has the potential to revolutionize modern medicine. With numerous clinical trials underway and the recent approval of the first genome editing therapy, the field is rapidly advancing. However, there is a consensus that clinical genome editing tools must be efficient, precise and safe. Current tools predominantly rely on host cell DNA repair pathways to complete the editing process, which poses a risk for unintended editing events. Host cell DNA repair independent, fully programmable editing tools represent a new class of genome editing technologies, significantly expanding the potential for delivering life-saving therapies to patients.
Genome editing tools that fulfil these characteristics are enzymes that recombine DNA. While there has been some success in engineering these enzymes to recognize and act on therapeutically relevant targets, their time-consuming and labour-intensive generation has hindered widespread implementation. DC-PGE aims to address this shortcoming. We have recently demonstrated that recombinase activity can be conditioned by internally fusing a zinc-finger DNA-binding domain into the enzyme's coding sequence, thereby making the enzyme activity contingent on zinc finger DNA binding. This pioneering discovery presents a genuine opportunity to develop next generation and best-in-class genome editing enzymes.
DC-PGE aims to i) build a platform to apply DNA-binding conditioned precision genome editing to DNA-recombining enzymes, ii) develop a pan-recombinase system for immediate targeting of virtually any genomic sequence and iii) apply the technology to correct the genetic cause of MECP2 duplication syndrome at nucleotide precision. DC-PGE will serve as a foundation for advancing innovative treatments and improving patient outcomes. Our unique approach lies in combining different disciplines and cutting-edge technologies with the potential to transform the field of genome editing and elevate it to the next level.
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.
This project's classification has been human-validated.
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.
This project's classification has been human-validated.
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)
MAIN PROGRAMME
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Topic(s)
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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.
Funding Scheme
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.
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
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
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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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.
01069 Dresden
Germany
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.