Stem Cell-Derived Spinal Cord Organoid Model: Temporal and Cell-Specific Analysis of Molecular Effects of a Risdiplam Analogue (N2.002)
To investigate the molecular and functional effects of a risdiplam-like compound (RIS-L) using a 3D human spinal cord organoid model derived from iPSCs of spinal muscular atrophy (SMA) type 1 patients and healthy controls, aiming to optimize treatment parameters and identify potential complementary targets.
Authors
- Andrea D’Angelo
- Giacomo P. Comi (ORCID: https://orcid.org/0000-0002-1383-5248)
- Delia Gagliardi (ORCID: https://orcid.org/0000-0001-8460-938X)
- Linda Ottoboni (ORCID: https://orcid.org/0000-0001-5412-7785)
- Megi Meneri (ORCID: https://orcid.org/0000-0002-3429-1809)
- Chiara Cordiglieri (ORCID: https://orcid.org/0000-0002-2013-0043)
- Simona Lodato (ORCID: https://orcid.org/0000-0001-6498-2590)
- Federica Rizzo (ORCID: https://orcid.org/0000-0002-7677-0330)
- Matteo Miotto (ORCID: https://orcid.org/0000-0003-3288-9011)
- Francesca Beatrice
- Stefania Corti
Institutions
- Humanitas University (IT)
- University of Milan (IT)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Ospedale Maggiore (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000208466
- Citations
- 1
- Primary Topic
- Chemical Synthesis and Analysis
- Type
- article
- Field-Weighted Citation Impact
- 0.81