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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: fnu n. 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.
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: fnu n. Int J Mol Sci. 2022 Oct 21;23(20):12693. doi: 10.3390/ijms232012693. Int J Mol Sci. 2022. PMID: 36293546 Free PMC article.