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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. Among authors: herreno pachon am. 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.
Bone Growth Induction in Mucopolysaccharidosis IVA Mouse.
Rintz E, Herreño-Pachón AM, Celik B, Nidhi F, Khan S, Benincore-Flórez E, Tomatsu S. Rintz E, et al. Among authors: herreno pachon am. Int J Mol Sci. 2023 Jun 8;24(12):9890. doi: 10.3390/ijms24129890. Int J Mol Sci. 2023. PMID: 37373036 Free PMC article.
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: pachon amh. Int J Mol Sci. 2022 Oct 21;23(20):12693. doi: 10.3390/ijms232012693. Int J Mol Sci. 2022. PMID: 36293546 Free PMC article.