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Page 1
Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy.
Piechnik M, Amendum PC, Sawamoto K, Stapleton M, Khan S, Fnu N, Álvarez V, Pachon AMH, Danos O, Bruder JT, Karumuthil-Melethil S, Tomatsu S. Piechnik M, et al. Among authors: stapleton m. Int J Mol Sci. 2022 Oct 21;23(20):12693. doi: 10.3390/ijms232012693. Int J Mol Sci. 2022. PMID: 36293546 Free PMC article.
Newborn screening of mucopolysaccharidoses: past, present, and future.
Arunkumar N, Langan TJ, Stapleton M, Kubaski F, Mason RW, Singh R, Kobayashi H, Yamaguchi S, Suzuki Y, Orii K, Orii T, Fukao T, Tomatsu S. Arunkumar N, et al. Among authors: stapleton m. J Hum Genet. 2020 Jul;65(7):557-567. doi: 10.1038/s10038-020-0744-8. Epub 2020 Apr 10. J Hum Genet. 2020. PMID: 32277174 Review.
Development of a newborn screening tool for mucopolysaccharidosis type I based on bivariate normal limits: Using glycosaminoglycan and alpha-L-iduronidase determinations on dried blood spots to predict symptoms.
Langan TJ, Jalal K, Barczykowski AL, Carter RL, Stapleton M, Orii K, Fukao T, Kobayashi H, Yamaguchi S, Tomatsu S. Langan TJ, et al. Among authors: stapleton m. JIMD Rep. 2020 Feb 10;52(1):35-42. doi: 10.1002/jmd2.12093. eCollection 2020 Mar. JIMD Rep. 2020. PMID: 32154058 Free PMC article.
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.
Taylor M, Khan S, Stapleton M, Wang J, Chen J, Wynn R, Yabe H, Chinen Y, Boelens JJ, Mason RW, Kubaski F, Horovitz DDG, Barth AL, Serafini M, Bernardo ME, Kobayashi H, Orii KE, Suzuki Y, Orii T, Tomatsu S. Taylor M, et al. Among authors: stapleton m. Biol Blood Marrow Transplant. 2019 Jul;25(7):e226-e246. doi: 10.1016/j.bbmt.2019.02.012. Epub 2019 Feb 14. Biol Blood Marrow Transplant. 2019. PMID: 30772512 Free PMC article. Review.
Critical review of current MPS guidelines and management.
Stapleton M, Hoshina H, Sawamoto K, Kubaski F, Mason RW, Mackenzie WG, Theroux M, Kobayashi H, Yamaguchi S, Suzuki Y, Fukao T, Tadao O, Ida H, Tomatsu S. Stapleton M, et al. Mol Genet Metab. 2019 Mar;126(3):238-245. doi: 10.1016/j.ymgme.2018.07.001. Epub 2018 Jul 7. Mol Genet Metab. 2019. PMID: 30143438 Review.
14 results