Project description
Gearing towards genome surgery
Synthetic genomes are chemically created piece by piece by scientists and then inserted into cells. Despite success with viral and bacterial genomes, synthesising a eukaryotic genome has proved to be challenging and assembly has so far been limited to a few chromosomes. The EU-funded RE-GENESis project is using gene editing methodologies to replace large parts of DNA in embryonic stem cells. With a focus on correcting the Duchenne muscular dystrophy gene, researchers have developed new DNA modification and replacement tools. RE-GENESis will not only contribute towards the understanding of complex eukaryotic genomes but also provide novel solutions to treat genetic diseases.
Objective
The assembly of functional synthetic mammalian genomes is the most ambitious biotechnological challenge of the current century. Synthetic genomics has generated remarkable results in viruses and bacteria, but the de novo assembly of eukaryotic genomes is still limited to a few small chromosomes. Indeed, efficient delivery and genome engineering strategies for systematic genome replacement and assembly in higher eukaryotic cells are still missing. With my project RE-GENESis, I intend to make an essential contribution toward a more comprehensive understanding on the mammalian genome through the synthetic replacement of recoded loci at megabase-scale. My main goal is to demonstrate the feasibility of synthetic genomics and genetic code reprogramming at the megabase-scale in mammalian cells, using mouse embryonic stem cells to identify efficient delivery vectors, genome editing strategies and recoding schemes. I will use CRISPR-Cas in combination with large vectors to find new genome surgery strategies for genome recoding and gene correction and exploit them to replace the 2.4 Mb DMD (Duchenne Muscular Dystrophy)/dystrophin gene as a landmark application. The developed delivery and DNA replacement strategies will be also readily available for genome editing therapeutic approaches in order to correct most of the DMD disease-related mutations in human stem cells. RE-GENESis will thus pave the way for the effective deployment of synthetic genomics in high eukaryotes, expanding our understanding of complex eukaryotic genomes, delivering new tools to modify and regulate mammalian DNA and providing innovative solutions to treat genetic diseases, epidemics and ageing. This highly original and multidisciplinary project combines the strength of CRISPR-Cas with the power of large delivery vectors to yield a project of excellent, innovative science that will exploit my expertise in genome editing while providing me extensive training in genome recoding and synthetic genomics.
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.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences clinical medicine surgery
- medical and health sciences medical biotechnology cells technologies stem cells
- medical and health sciences basic medicine neurology muscular dystrophies duchenne muscular dystrophy
- natural sciences biological sciences genetics genomes eukaryotic genomes
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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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H2020-EU.1.3. - EXCELLENT SCIENCE - Marie Skłodowska-Curie Actions
MAIN PROGRAMME
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H2020-EU.1.3.2. - Nurturing excellence by means of cross-border and cross-sector mobility
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Topic(s)
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.
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.
MSCA-IF-EF-ST - Standard EF
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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) H2020-MSCA-IF-2019
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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.
SN2 1FL SWINDON
United Kingdom
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.