Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts

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Authors: Ilaria Signoria, Maria M. Zwartkruis, Lotte Geerlofs, Elena Perenthaler, Kiterie M.E. Faller, Rachel James, Harriet McHale-Owen, Jared W. Green, Joris Kortooms, Sophie H. Snellen, Fay-Lynn Asselman, Thomas H. Gillingwater, Gabriella Viero, Renske I. Wadman, W. Ludo van der Pol, Ewout J.N. Groen

Journal title: biorxiv

Journal publisher: bioRxiv

Published year: 2024

DOI identifier: 10.1101/2024.07.24.604892

ISSN: 2692-8205