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Matteo Miotto

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Articles

Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids - Nature Communications

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By Irene Faravelli, Paola Rinchetti, Monica Tambalo, Illia Simutin, Lisa Mapelli, Sara Mancinelli, Matteo Miotto, Mafalda Rizzuti, Chiara Cordiglieri, Paolo Kunderfranco, Egidio D’Angelo, Serge Przedborski, Monica Nizzardo, Simona Lodato, Giacomo P. Comi, Francesca Beatrice
| Nature Verified
Abstract Spinal muscular atrophy (SMA) is a severe neurological disease caused by mutations in the SMN1 gene, characterized by early onset and degeneration of lower motor neurons. Understanding early neurodevelopmental defects in SMA is crucial for optimizing therapeutic interventions. Using spinal cord and cerebral organoids generated from multiple SMA type 1 male donors, we revealed widespread disease mechanisms beyond motor neuron degeneration.
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Neurodevelopmental origins of structural and psychomotor defects in CXCR4-linked primary immunodeficiency

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By Giulia Demenego, Sara Mancinelli, Matteo Miotto, Marco Cremonesi, Irene Corradini, Eugene Kim, Katarina Ilic, Luca Pellegrino, Raffaele Badolato, Roberto Rusconi, Antonella Borreca, Rosalba Olga Proce, Vanessa Aragona, Laura Zucchelli, Edoardo Fraviga
| Cell Press Verified
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