Transferring autonomous and non autonomous cell degeneration 3D models between EU and USA for development of effective therapies for neurodegenerative diseases (ND)

Livrables

Report for the on-site training provided and usage of the online system every 6 months

We will report for the onsite training provided and usage of the online system

Evaluation of neuroprotective abilities of candidate genes in an in vitro model.

We will assess gene expression profile and identify candidate causal MRs and shared molecular pathways between NDs.

3D models generated from 4 different sources

Patient specific minibrain and spinal cordorganoids generated from iPSCs of ALS SMA FTD and PD will be characterized

Pre-clinical protocols for AAV gene delivery

We will establish protocols to over-express or knock-down genes of interest by using adeno-associated virus (AAV).

Identification of neuropathological markers

We will identify genes responsible for selective cell death and potential targets to modify ND disease progression

Publications

Human 3D cell culture systems to model and elucidate spinal muscular atrophy pathology and treatment

Auteurs: *S. CORTI1, I. FARAVELLI1, P. RINCHETTI1, S. MANCINELLI2, L. MAPELLI3, G. FOROTTI4, M. RIZZUTI4, C. CORDIGLIERI5, L. CALANDRIELLO4, S. TAMANINI1, N. BRESOLIN1, G. P. COMI1, S. LODATO2, M. NIZZARDO1
Publié dans: 2019
Éditeur: SFN

CSF transplantation of a specific iPSC-derived neural stem cell subpopulation ameliorates the disease phenotype in a mouse model of spinal muscular atrophy with respiratory distress type 1.

Auteurs: Forotti G1, Nizzardo M1, Bucchia M2, Ramirez A2, Trombetta E3, Gatti S4, Bresolin N5, Comi GP5, Corti S6.
Publié dans: Experimental Neurology, 2019, ISSN 0014-4886
Éditeur: Academic Press
DOI: 10.1016/j.expneurol.2019.113041

iPSCs-Based Neural 3D Systems: A Multidimensional Approach for Disease Modeling and Drug Discovery

Auteurs: Gianluca Costamagna, Luca Andreoli, Stefania Corti, Irene Faravelli
Publié dans: Cells, Numéro 8/11, 2019, Page(s) 1438, ISSN 2073-4409
Éditeur: Multidisciplinary Digital Publishing Institute
DOI: 10.3390/cells8111438

Disease Modeling and Therapeutic Strategies in CMT2A: State of the Art

Auteurs: Kordelia Barbullushi, Elena Abati, Federica Rizzo, Nereo Bresolin, Giacomo P. Comi, Stefania Corti
Publié dans: Molecular Neurobiology, Numéro 56/9, 2019, Page(s) 6460-6471, ISSN 0893-7648
Éditeur: Humana Press, Inc.
DOI: 10.1007/s12035-019-1533-2

Livrables

Publications

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