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Content archived on 2024-05-24

Development and application of chromosome-based gene transfer vectors for cell therapy

Objective

Human minichromosomes (MCs) to be employed in the context of a cell-based therapy of lung diseases will be constructed using techniques, which guarantee the retention of the structural features of natural chromosomes. MCs purified by flow sorting and carrying marker (e.g. GFP) or therapeutic (CFTR) genes will be transferred into human respiratory epithelial cells in vitro (artificial lungs) and in vivo (airway xenografts) settings using both microcells and novel transfection techniques (multi-domain vectors comprised of polycations, receptor ligand moieties and nuclear localisation signals). Improved CFTR-MCs will be introduced in cystic fibrosis (CF) cells or epithelia and complementation of CF defects will be evaluated. MCs will be used to generate chimeric animals, and transferred into stem/progenitor cells for the purpose of grafting regenerating lung epithelium in injury mouse models.

Call for proposal

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Coordinator

FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR - ISTITUTO DI RICOVERO E CURA A CARATTERE SCIENTIFICO
EU contribution
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Address
Via Olgettina 60 HSR Parco Scientifico
20132 MILANO
Italy

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Total cost
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Participants (5)