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Med (New York, N.Y.)|April 12, 2025
AAV gene therapy for mucopolysaccharidosesShaukat Khan, Yasuhiko Ago, Shunji TomatsuMolecular Genetics and Metabolism Reports|January 25, 2021
Novel GYS2 mutations in a Japanese patient with glycogen storage disease type 0aHiroyuki Iijima, Yasuhiko Ago, Ryoji Fujiki, et al.International Journal of Molecular Sciences|January 23, 2024
Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative TherapyYasuhiko Ago, Estera Rintz, Krishna Sai Musini, et al.Molecular Genetics & Genomic Medicine|October 8, 2021
Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacyHui Chen, Shaukat Khan, Betul Celik, et al.International Journal of Molecular Sciences|March 14, 2026
AAV Gene Therapy for MPS IVA with Induction of Immune Tolerance via Oral Administration of Epitope Peptides of N-Acetylgalactosamine-6-sulfate SulfataseSampurna Saikia, Yasuhiko Ago, Fnu Nidhi, et al.International Journal of Molecular Sciences|May 17, 2020
Evading the AAV Immune Response in MucopolysaccharidosesMatthew Piechnik, Kazuki Sawamoto, Hidenori Ohnishi, et al.Gene|April 27, 2018
Intronic antisense Alu elements have a negative splicing effect on the inclusion of adjacent downstream exonsMina Nakama, Hiroki Otsuka, Yasuhiko Ago, et al.Journal of Human Genetics|November 6, 2018
Recent advances in understanding beta-ketothiolase (mitochondrial acetoacetyl-CoA thiolase, T2) deficiencyToshiyuki Fukao, Hideo Sasai, Yuka Aoyama, et al.JIMD Reports|August 9, 2019
A rare PHKA2 variant (p.G991A) identified in a patient with ketotic hypoglycemiaYasuhiko Ago, Hideo Sugie, Tokiko Fukuda, et al.International Journal of Molecular Sciences|January 8, 2023
Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential TargetsAndrés Felipe Leal, Eliana Benincore-Flórez, Estera Rintz, et al.Pageof 2