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 V Kortooms, Sophie H. Snellen, Fay‐Lynn Asselman, Thomas Henry Gillingwater, Gabriella Viero, Renske I. Wadman, W. Ludo van der Pol, Ewout J. N. Groen
The paper analyzes responses to SMN2 splice-modifying treatments in SMA fibroblasts, finding variability in treatment efficacy based on patient-specific factors.
Molecular Therapy — Methods & Clinical Development · Nov 13, 2024Peer-reviewedMolecular Biology