Descrizione del progetto
Un nuovo fattore neurotrofico per il morbo di Parkinson
Il morbo di Parkinson (PD, Parkinson’s Disease) colpisce milioni di persone in tutto il mondo e, considerato l’invecchiamento demografico, tale cifra è destinata a raddoppiare entro il 2030. Nonostante anni di ricerche, non esiste una terapia per il PD. Il progetto BMPARK, finanziato dall’UE, sta indagando il potenziale della proteina morfogenetica dell’osso 2 (BMP2, Bone Morphogenetic Protein 2) come fattore neurotrofico, per proteggere i neuroni dopaminergici del mesencefalo nel cervello di pazienti affetti da PD. Utilizzando un modello di ratto della malattia, gli scienziati valuteranno l’efficacia della BMP2 nell’arrestare la perdita progressiva di neuroni dopaminergici e nel migliorare la funzione motoria. I risultati del progetto decifreranno la modalità di azione della BMP2 e contribuiranno allo sviluppo futuro di strategie cliniche per il PD.
Obiettivo
Parkinson’s Disease (PD) is a progressive neurodegenerative disease. PD affects more than 6 million people worldwide while 1.2 million people have PD in Europe. For the European economy PD has a total socioeconomic cost of €13.9 billion per annum. With the aging demographic the number of individuals affects by PD are projected to double by 2030 highlight the need for disease modifying therapies. The neuropathological hallmarks of PD are the progressive loss of dopaminergic neurons in a region of the brain known as the substantia nigra in the midbrain, and the accumulation of intraneuronal inclusions called Lewy bodies and lewy neurite that consist predominantly of a protein called α-synuclein. Despite over half a century of investigation, there is no disease modifying therapy for PD. I propose that a protein called bone morphogenetic protein (BMP)2 can protect midbrain dopaminergic neurons in the PD brain. BMP2 is distinct from other neurotrophic factors used in the clinical trials to date in that it has a different signalling mechanism that can be targeted to protect dopaminergic neurons. Here I will assess the neuroprotective efficacy of viral vectors carrying the BMP2 transgene in the rat α-synuclein model of PD. I will determine if this molecular therapy can protect dopamine neurons and their axons against α-synuclein induced degeneration to maintain brain dopamine levels and improve motor function. To do this, I will use a multidisciplinary approach which will be paralleled by intensive training and career development opportunities to enable me to make a transformative contribution to knowledge and to achieve professional maturity and independence. In this way, this work has direct relevance for the future development of neurotrophic factors for clinical use in PD and the extensive dissemination and communication plans to target all stakeholders will ensure both societal and scientific impact in an European context and also for individuals with PD.
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MSCA-IF - Marie Skłodowska-Curie Individual Fellowships (IF)Coordinatore
T12 YN60 Cork
Irlanda