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...

وصف كامل

التفاصيل البيبلوغرافية
المؤلفون الرئيسيون:	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
التنسيق:	مقال
اللغة:	English
منشور في:	Elsevier 2024-12-01
سلاسل:	Molecular Therapy: Methods & Clinical Development
الموضوعات:	spinal muscular atrophy gene therapy disease models splice-modifiers personalized medicine
الوصول للمادة أونلاين:	http://www.sciencedirect.com/science/article/pii/S2329050124001955

الانترنت

http://www.sciencedirect.com/science/article/pii/S2329050124001955

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

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