Project LIMONE – Lipid-based nanoparticles to reach the motor neuron
Description of the project: Spinal muscular atrophy (SMA) is a genetic disease characterized by the degeneration of motor neurons and progressive atrophy of muscles of limbs, trunk and respiratory system. SMA is the main cause of infancy death and affects 1 per 10000 people worldwide. Nusinersen is the first drug approved for the treatment of SMA (2016) and, due to its inability in crossing the blood brain barrier (BBB), it is administered intratechally; besides the side effects due to the route of administration, Nusinersen can also cause thrombocytopenia, hydrocephalus and renal toxicity.
Aim of the project: LIMONE aims at developing liposomes able to encapsulate Nusinersen and deliver it to the motor neuron (MN) crossing the BBB. The attainment of these systems would greatly improve the therapy, both in terms of benefit for the patients and costs for the Sanitary System.
Results: The project aims at implementing the results of the project LIPOBARR (Gruppi di Ricerca 2018), in which liposomes able to cross the BBB have been developed. The goal is to confer to the liposomes specificity towards MN, a key feature that, by decreasing side effects, will represent a strong improvement of the current formulation.
- CNR-ISB (Capofila), Francesca Ceccacci, Giovanna Mancini
- CNR-IBBC, Maria Laura Falchetti
- CNR-NANOTEC, Valentina Arima
- Dipartimento di Chimica Università di Roma “La Sapienza”: Anita Scipioni
- Dipartimento DIBAF, Università della Tuscia: Stefano Borocci
Funding institutions: FESR Fondo Europeo di Sviluppo Regionale - Programma Operativo regionale del Lazio – Programmazione 2014-2020. Avviso Pubblico: “Progetti di Gruppi di ricerca 2020”
Funding: 149.689 €
Project Duration: 15/04/2021 – 15/04/2023