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The landscape of CRISPR/Cas9 for inborn errors of metabolism.
Leal AF, Fnu N, Benincore-Flórez E, Herreño-Pachón AM, Echeverri-Peña OY, Alméciga-Díaz CJ, Tomatsu S. Leal AF, et al. Mol Genet Metab. 2023 Jan;138(1):106968. doi: 10.1016/j.ymgme.2022.106968. Epub 2022 Dec 7. Mol Genet Metab. 2023. PMID: 36525790 Free PMC article. Review.
Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.
Tomatsu S, Sawamoto K, Alméciga-Díaz CJ, Shimada T, Bober MB, Chinen Y, Yabe H, Montaño AM, Giugliani R, Kubaski F, Yasuda E, Rodríguez-López A, Espejo-Mojica AJ, Sánchez OF, Mason RW, Barrera LA, Mackenzie WG, Orii T. Tomatsu S, et al. Drug Des Devel Ther. 2015 Apr 1;9:1937-53. doi: 10.2147/DDDT.S68562. eCollection 2015. Drug Des Devel Ther. 2015. PMID: 25897204 Free PMC article. Review.
75 results