Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets von Leal, Andrés Felipe, Benincore-Flórez, Eliana, Rintz, Estera, Herreño-Pachón, Angélica María, Celik, Betul, Ago, Yasuhiko, Alméciga-Díaz, Carlos Javier, Tomatsu, Shunji

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Bone Growth Induction in Mucopolysaccharidosis IVA Mouse von Rintz, Estera, Herreño-Pachón, Angélica María, Celik, Betul, Nidhi, Fnu, Khan, Shaukat, Benincore-Flórez, Eliana, Tomatsu, Shunji

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Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice von Rintz, Estera, Celik, Betul, Fnu, Nidhi, Herreño-Pachón, Angélica María, Khan, Shaukat, Benincore-Flórez, Eliana, Tomatsu, Shunji

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The landscape of CRISPR/Cas9 for inborn errors of metabolism von Leal, Andrés Felipe, Fnu, Nidhi, Benincore-Flórez, Eliana, Herreño-Pachón, Angelica María, Echeverri-Peña, Olga Yaneth, Alméciga-Díaz, Carlos Javier, Tomatsu, Shunji

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Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches von Leal, Andrés Felipe, Herreno-Pachón, Angelica María, Benincore-Flórez, Eliana, Karunathilaka, Amali, Tomatsu, Shunji

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Molecular therapy and nucleic acid adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice von Rintz, Estera, Celik, Betul, Fnu, Nidhi, Herreño-Pachón, Angélica María, Khan, Shaukat, Benincore-Flórez, Eliana, Tomatsu, Shunji

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Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy von Piechnik, Matthew, Amendum, Paige C, Sawamoto, Kazuki, Stapleton, Molly, Khan, Shaukat, Fnu, Nidhi, Álvarez, Victor, Pachon, Angelica Maria Herreño, Danos, Olivier, Bruder, Joseph T, Karumuthil-Melethil, Subha, Tomatsu, Shunji

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Transición epitelio mesénquima: de lo molecular a lo fisiológico von Troncoso, Daniela, Madariaga Perpiñan, Ithzayana, Aldana Mancera, Sergio Andrés, Herreño Pachón, Angélica María, Chaparro Ramírez, Viviana Paola, Molina Camargo, Mónica Lorena, Rey Vargas, Laura, Ramírez Rodríguez, Andrea Carolina, Montoya Estupiñan, Christian Fernando, Valderrama Álvarez, Andrea, Cañas Arboleda, Alejandra, Rojas Moreno, Adriana Patricia

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