Biblio

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Journal Article

Rizzo F, Bono S, Ruepp MDavid, Salani S, Ottoboni L, Abati E, Melzi V, Cordiglieri C, Pagliarani S, De Gioia R, et al. Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A. Cell Mol Life Sci. 2023;80(12):373.

Forotti G, Nizzardo M, Bucchia M, Ramirez A, Trombetta E, Gatti S, Bresolin N, Comi GPietro, Corti S. 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. Exp Neurol. 2019:113041.

Nizzardo M, Simone C, Rizzo F, Salani S, Dametti S, Rinchetti P, Del Bo R, Foust K, Kaspar BK, Bresolin N, et al. Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model. Sci Adv. 2015;1(2):e1500078.

Gamboa ALucia Cuad, Benfante R, Nizzardo M, Bachetti T, Pelucchi P, Melzi V, Arzilli C, Peruzzi M, Reinbold RA, Cardani S, et al. Generation of two hiPSC lines (UMILi027-A and UMILi028-A) from early and late-onset Congenital Central hypoventilation Syndrome (CCHS) patients carrying a polyalanine expansion mutation in the PHOX2B gene. Stem Cell Res. 2022;61:102781.

Nizzardo M, Simone C, Rizzo F, Ruggieri M, Salani S, Riboldi G, Faravelli I, Zanetta C, Bresolin N, Comi GP, et al. Minimally invasive transplantation of iPSC-derived ALDHhiSSCloVLA4+ neural stem cells effectively improves the phenotype of an amyotrophic lateral sclerosis model. Hum Mol Genet. 2013.

Abati E, Bresolin N, Comi GPietro, Corti S. Preconditioning and Cellular Engineering to Increase the Survival of Transplanted Neural Stem Cells for Motor Neuron Disease Therapy. Mol Neurobiol. 2018.

Ripolone M, Lucchini V, Ronchi D, Fagiolari G, Bordoni A, Fortunato F, Mondello S, Bonato S, Meregalli M, Torrente Y, et al. Purkinje cell COX deficiency and mtDNA depletion in an animal model of spinocerebellar ataxia type 1. J Neurosci Res. 2018;96(9):1576-1585.

Rizzuti M, Melzi V, Brambilla L, Quetti L, Sali L, Ottoboni L, Meneri M, Ratti A, Verde F, Ticozzi N, et al. Shaping the Neurovascular Unit Exploiting Human Brain Organoids. Mol Neurobiol. 2024.