Survival of Motor Neuron 2 Protein

Displaying 1 - 3 of 3CSV
Sutherland, C. S., Schneider, S., Ribero, V. A., Simpson, A., Kokaliaris, C., Scalco, R. S., Guittari, C. J., Gorni, K., De Vivo, D. C., Martens, W. B., & Karrer, T. M. (2025). The influence of genotype on the natural history of types 1 - 3 spinal muscular atrophy. Neuromuscular Disorders, 47, 105270. https://doi.org/10.1016/j.nmd.2024.105270
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Tizzano, E. F., Quijano-Roy, S., Servais, L., Parsons, J. A., Aharoni, S., Lakhotia, A., Finkel, R. S., Mercuri, E., Kirschner, J., De Vivo, D. C., Saito, K., Raju, D., Benguerba, K., Dabbous, O., Mumneh, N., Reyna, S. P., & Faulkner, E. (2024). Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec. European Journal of Paediatric Neurology, 53, 18–24. https://doi.org/10.1016/j.ejpn.2024.08.006
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Recinos, Y., Ustianenko, D., Yeh, Y.-T., Wang, X., Jacko, M., Yesantharao, L. V., Wu, Q., & Zhang, C. (2024). CRISPR-dCas13d-based deep screening of proximal and distal splicing-regulatory elements. Nature Communications, 15(1). https://doi.org/10.1038/s41467-024-47140-8
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