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Base editing corrects the common Salla disease SLC17A5 c.115C>T variant.
Harb JF, Christensen CL, Kan SH, Rha AK, Andrade-Heckman P, Pollard L, Steet R, Huang JY, Wang RY. Harb JF, et al. Among authors: christensen cl. Mol Ther Nucleic Acids. 2023 Aug 26;34:102022. doi: 10.1016/j.omtn.2023.08.024. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 37727271 Free PMC article.
Progranulin as a Novel Factor in Gaucher Disease.
Choy FYM, Christensen CL. Choy FYM, et al. Among authors: christensen cl. EBioMedicine. 2016 Nov;13:13-14. doi: 10.1016/j.ebiom.2016.11.006. Epub 2016 Nov 5. EBioMedicine. 2016. PMID: 27836396 Free PMC article. No abstract available.