Project description
Treating cystic fibrosis using nanogels
Cystic fibrosis is one of the most serious genetic lung diseases. It is characterised by the build-up of thick mucus in the lungs, leading to infection and breathing difficulties. While advanced modulator therapies have significantly reduced mortality rates, there is still no cure. Supported by the Marie Skłodowska-Curie Actions programme, the CRISPRGels project aims to develop non-viral gene delivery systems for the treatment of cystic fibrosis based on nanogel-mediated controlled release of the CRISPR/Cas9 complex. It will methodically examine the behaviour and interactions of the nanogels in cystic fibrosis patient-derived sputum and bronchial epithelial models. Then it will validate the most effective nanogels on a pre-industrial scale.
Objective
Cystic fibrosis (CF) is the most common genetic lung disease (monogenic autosomal recessive disorder) worldwide and currently there is no cure. The emergence of powerful gene editing tools like CRISPR-Cas (“clustered regularly interspaced short palindromic repeats/associated protein 9”), now provides the opportunity to correct the disease-causing mutations which can potentially cure CF. The present research project proposes the development of core-multishell nanogels (NGs) yielding efficient transmucosal delivery of CRISPR-based gene editing tools which can address the challenges for treatment of CF at European level. This project brings together a talented, highly motivated and young Researcher (Dr. Krishan Kumar) with background in polymer chemistry (phase transition temperatures, understanding structure of colloids, bio-macromolecular interactions) and protein science (structural stability, immobilization, enzymatic activity); and an innovative institute POLYMAT in developing biomedical solutions for treating CF diseases based on nanogels mediated controlled release of CRISPR/Cas9 complex. CRISPRGels will focus on advancing the young researcher’s career through development and manufacture stages of non-viral gene delivery systems facilitating the curative gene editing approaches for treatment of CF patients. The host supervisor Prof. Marcelo Calderón is globally recognized researcher working in area of transmucosal delivery using nanogels (NGs). The behavior and interactions of the NGs will be systematically investigated in CF patient-derived sputum and CF bronchial epithelial models. Most efficient NGs will be validated on pre-industrial scale. Multidisciplinary expertise required for CRISPRGels will be complemented by highly experienced research teams Responsive Polymer Therapeutics Group (RPTG), Translational Organ Models Group (TOMG) and i+Med company.
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
- natural sciences biological sciences genetics mutation
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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.1.2 - Marie Skłodowska-Curie Actions (MSCA)
MAIN PROGRAMME
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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.
HORIZON-TMA-MSCA-PF-EF - HORIZON TMA MSCA Postdoctoral Fellowships - European Fellowships
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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) HORIZON-MSCA-2022-PF-01
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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.
48940 Leioa
Spain
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.