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Rational Nanoparticle Design for Efficient Transmucosal CRISPR Delivery in Cystic Fibrosis – “CRISPRGels”

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.

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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-2022-PF-01

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Coordinator

UNIVERSIDAD DEL PAIS VASCO/ EUSKAL HERRIKO UNIBERTSITATEA
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.

€ 206 641,20
Address
BARRIO SARRIENA S N
48940 LEIOA
Spain

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Region
Noreste País Vasco Gipuzkoa
Activity type
Higher or Secondary Education Establishments
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Total cost

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.

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Partners (3)

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