VEXOSOMES as stealth natural nanosystems to treat monogenic diseases
Tesi esterna in azienda
Riferimenti VALENTINA ALICE CAUDA
Riferimenti esterni Prof. Marta Guerra Rebollo
Prof. Cristina Fornaguera
Institut Quimico de SArrža- Universidad Ramon Lull, Barcelona- Spain
Gruppi di ricerca AA - Materials and Processes for Micro and Nano Technologies
Tipo tesi SPERIMENTALE
Descrizione Currently, many monogenic diseases therapies are based on the use of adeno-associated viral vectors (AAV). However, these can be prematurely neutralized by the patient immune system after the first administration. In this context, an interesting improvement could be their coating with lipid components, such as extracellular vesicles (EVs). EVs could be advantageous in terms of being non-immunogenic and naturally targeted to the cell lineage they come from. Therefore, VEXOSOMES, this is, engineered AAVs coated with EVs could become the next generation therapies of currently monogenic orphan diseases, such as muscular dystrophies.
The aim of this project is optimizing the efficient coating of AAVs with EVs to achieve they can be in vivo administered and arrive to their target organs without being previously cleared up by neutralizing antibodies. This objective can be split up into: 1) Set up the methodology for the most efficient coating method. 2) In vitro study the tropism shift of the AAVs thanks to the EV coating. 3) In vivo studies of biodistribution of the engineered VEXOSOMES.
Methodologies: nanocharacterization, celular biology, cell culture, exosome isolation and characterization, molecular biology, cell cytometry, confocal microscopy.
Directors : Dr. Cristina Fornaguera and Dr. Marta Guerra
Conoscenze richieste nanoparticelle, caratterizazzione di nanoparticelle, basi di biologia e chimica, scienza dei materiali
Note Methodologies: nanocharacterization, celular biology, cell culture, exosome isolation and characterization, molecular biology, cell cytometry, confocal microscopy.
Scadenza validita proposta 15/12/2023 PROPONI LA TUA CANDIDATURA