Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy
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
Published in International journal of molecular sciences (21.10.2022)
Published in International journal of molecular sciences (21.10.2022)
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The landscape of CRISPR/Cas9 for inborn errors of metabolism
Leal, Andrés Felipe, Fnu, Nidhi, Benincore-Flórez, Eliana, Herreño-Pachón, Angelica María, Echeverri-Peña, Olga Yaneth, Alméciga-Díaz, Carlos Javier, Tomatsu, Shunji
Published in Molecular genetics and metabolism (01.01.2023)
Published in Molecular genetics and metabolism (01.01.2023)
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Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches
Leal, Andrés Felipe, Herreno-Pachón, Angelica María, Benincore-Flórez, Eliana, Karunathilaka, Amali, Tomatsu, Shunji
Published in International journal of molecular sciences (20.02.2024)
Published in International journal of molecular sciences (20.02.2024)
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Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice
Rintz, Estera, Celik, Betul, Fnu, Nidhi, Herreño-Pachón, Angélica María, Khan, Shaukat, Benincore-Flórez, Eliana, Tomatsu, Shunji
Published in Molecular therapy. Nucleic acids (11.06.2024)
Published in Molecular therapy. Nucleic acids (11.06.2024)
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Glycosaminoglycans in mucopolysaccharidoses and other disorders
Khan, Shaukat A, Nidhi, Fnu, Leal, Andrés Felipe, Celik, Betul, Herreño-Pachón, Angelica María, Saikia, Sampurna, Benincore-Flórez, Eliana, Ago, Yasuhiko, Tomatsu, Shunji
Published in Advances in clinical chemistry (01.01.2024)
Published in Advances in clinical chemistry (01.01.2024)
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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
Rintz, Estera, Celik, Betul, Fnu, Nidhi, Herreño-Pachón, Angélica María, Khan, Shaukat, Benincore-Flórez, Eliana, Tomatsu, Shunji
Published in Molecular therapy. Nucleic acids (11.06.2024)
Published in Molecular therapy. Nucleic acids (11.06.2024)
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Transición epitelio mesénquima: de lo molecular a lo fisiológico
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
Published in Universitas médica (01.10.2017)
Published in Universitas médica (01.10.2017)
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