Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids
Year: 2025
Type: article
Language: en
Topic: Neurogenetic and Muscular Disorders Research
Authors and affiliations:
raw_author_name: Irene Faravelli
raw_affiliation_strings: Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA, USA
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy
raw_author_name: Paola Rinchetti
raw_affiliation_strings: Department of Systems Biology, Columbia University, New York, NY, USA
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy
raw_author_name: Monica Tambalo
raw_affiliation_strings: Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, MI, Italy
raw_author_name: Illia Simutin
raw_affiliation_strings: Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, MI, Italy
raw_author_name: Lisa Mapelli
raw_affiliation_strings: Department of Brain and Behavioral Sciences, University of Pavia, Pavia, Italy
raw_author_name: Sara Mancinelli
raw_affiliation_strings: Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, MI, Italy
IRCCS Humanitas Clinical and Research Hospital, Rozzano-Milan, Italy
raw_author_name: Matteo Miotto
raw_affiliation_strings: IRCCS Humanitas Clinical and Research Hospital, Rozzano-Milan, Italy
raw_author_name: Mafalda Rizzuti
raw_affiliation_strings: Department of Neurosciences, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Neurology, Milan, Italy
raw_author_name: Andrea D’Angelo
raw_author_name: Chiara Cordiglieri
raw_affiliation_strings: Istituto Nazionale di Genetica Molecolare "Romeo ed Enrica Invernizzi", Milan, Italy
raw_author_name: Giulia Forotti
raw_affiliation_strings: Department of Neurosciences, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Neurology, Milan, Italy
raw_author_name: Clelia Peano
raw_affiliation_strings: Human Technopole, Milan, Italy
Institute of Genetic and Biomedical Research, UoS of Milan, National Research Council, Rozzano, MI, Italy
raw_author_name: Paolo Kunderfranco
raw_affiliation_strings: Bioinformatics Unit, IRCCS Humanitas Clinical and Research Hospital, Rozzano-Milan, Italy
raw_author_name: Luca Calandriello
raw_affiliation_strings: Department of Neurosciences, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Neurology, Milan, Italy
raw_author_name: Giacomo P. Comi
raw_affiliation_strings: Department of Neurosciences, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Neurology, Milan, Italy
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy
raw_author_name: Elvezia Paraboschi
raw_affiliation_strings: Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, MI, Italy
IRCCS Humanitas Clinical and Research Hospital, Rozzano-Milan, Italy
raw_author_name: Eleonora Pali
raw_affiliation_strings: Department of Brain and Behavioral Sciences, University of Pavia, Pavia, Italy
raw_author_name: Francesca Beatrice
raw_affiliation_strings: Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy
raw_author_name: Egidio D’Angelo
raw_affiliation_strings: Digital Neuroscience Center, IRCCS Mondino Foundation, Pavia, Italy
raw_author_name: Serge Przedborski
raw_affiliation_strings: Center for Motor Neuron Biology and Disease, Columbia University, New York, NY, USA
Department of Neurology, Columbia University, New York, NY, USA
Department of Neuroscience, Columbia University, New York, NY, USA
Department of Pathology and Cell Biology, Columbia University, New York, NY, USA
raw_author_name: Monica Nizzardo
raw_affiliation_strings: Department of Neurosciences, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Neurology, Milan, Italy
raw_author_name: Simona Lodato
raw_affiliation_strings: Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, MI, Italy. simona.lodato@hunimed.eu
IRCCS Humanitas Clinical and Research Hospital, Rozzano-Milan, Italy. simona.lodato@hunimed.eu
raw_author_name: Stefania Corti
raw_affiliation_strings: Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy. stefania.corti@unimi.it
Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, SSD Neuromuscular and rare diseases, Milan, Italy. stefania.corti@unimi.it