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Overexpression of adapted U1snRNA in patients' cells to correct a 5' splice site mutation in propionic acidemia |
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| Authors: | Sánchez-Alcudia Rocío Pérez Belén Pérez-Cerdá Celia Ugarte M Desviat Lourdes R |
| Institution: | Centro de Diagnóstico de Enfermedades Moleculares, Centro de Biología Molecular Severo Ochoa, UAM-CSIC, Universidad Autónoma de Madrid, Madrid, Spain. |
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