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Based on these properties, AAV9 was investigated in mice models of spinal muscular atrophy (SMA), a monogenic disease characterized by the degeneration of the spinal motor neurons, with positive results in motor neuron survival, reversal of the phenotype and increased survival (Dominguez et al

Balducci S, Iacobellis G, Parisi L, Di Biase N, Calandriello E, Leonetti F, et al

Nat Commun 10:1686 Morales C, Losada A (2018) Establishing and dissolving cohesion during the vertebrate cell cycle

Oncogene 43 (27), 21042114

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