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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Journal Article
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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Journal Article
Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice
Leal, Andrés Felipe, Celik, Betul, Fnu, Nidhi, Khan, Shaukat, Tomatsu, Shunji, Alméciga-Díaz, Carlos Javier
Published in Molecular therapy. Methods & clinical development (14.12.2023)
Published in Molecular therapy. Methods & clinical development (14.12.2023)
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Journal Article
lentiviral gene therapy for mucopolysaccharidosis type IVA
Celik, Betul, Rintz, Estera, Nidhi, Fnu, Khan, Shaukat, Tomatsu, Shunji
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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Journal Article
Ex vivo lentiviral gene therapy for mucopolysaccharidosis type IVA
Celik, Betul, Rintz, Estera, Nidhi, Fnu, Khan, Shaukat, Tomatsu, Shunji
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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Journal Article
Lentiviral gene therapy for mucopolysaccharidosis type IVA
Celik, Betul, Rintz, Estera, Nidhi, Fnu, Khan, Shaukat, Tomatsu, Shunji
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
Assessment of an iron oxide-coupled CRISPR/nCas9 gene editing in a mucopolysaccharidosis type IVA mouse model: An update and future perspectives
Leal, Andres F., Celik, Betul, Nidhi, Fnu, Khan, Shaukat, Tomatsu, Shunji, Alméciga-Díaz, Carlos Javier
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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Journal Article
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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Journal Article
Glucosamine amends CNS pathology in mucopolysaccharidosis IIIC mouse expressing misfolded HGSNAT
Pan, Xuefang, Taherzadeh, Mahsa, Bose, Poulomee, Heon-Roberts, Rachel, Nguyen, Annie L A, Xu, TianMeng, Pará, Camila, Yamanaka, Yojiro, Priestman, David A, Platt, Frances M, Khan, Shaukat, Fnu, Nidhi, Tomatsu, Shunji, Morales, Carlos R, Pshezhetsky, Alexey V
Published in The Journal of experimental medicine (01.08.2022)
Published in The Journal of experimental medicine (01.08.2022)
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Journal Article
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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Journal Article
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 (2024)
Published in Advances in clinical chemistry (2024)
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