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

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*

*Corresponding author for this work

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Biochemistry, Genetics and Molecular Biology