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

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

The availability of three therapies for the neuromuscular disease spinal muscular atrophy (SMA) highlights the need to match patients to the optimal treatment. Two of these treatments (nusinersen and risdiplam) target splicing of SMN2, but treatment outcomes vary from patient to patient. An incomple...

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Main 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
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
spinal muscular atrophy
gene therapy
disease models
splice-modifiers
personalized medicine
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124001955
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