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Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.
Chu SN, Soupene E, Sharma D, Sinha R, McCreary T, Hernandez B, Shen H, Wienert B, Bowman C, Yin H, Lesch BJ, Jia K, Romero KA, Kostamo Z, Zhang Y, Tran T, Cordero M, Homma S, Hampton JP, Gardner JM, Conklin BR, MacKenzie TC, Sheehan VA, Porteus MH, Cromer MK. Chu SN, et al. Among authors: mackenzie tc. Cell Rep. 2025 Jan 3;44(1):115141. doi: 10.1016/j.celrep.2024.115141. Online ahead of print. Cell Rep. 2025. PMID: 39754719 Free article.
Large Differences in Small RNA Composition Between Human Biofluids.
Godoy PM, Bhakta NR, Barczak AJ, Cakmak H, Fisher S, MacKenzie TC, Patel T, Price RW, Smith JF, Woodruff PG, Erle DJ. Godoy PM, et al. Among authors: mackenzie tc. Cell Rep. 2018 Oct 30;25(5):1346-1358. doi: 10.1016/j.celrep.2018.10.014. Cell Rep. 2018. PMID: 30380423 Free PMC article.
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namuduri SR, Borges B, Kirn G, Valencia J, Lopez ME, Lui JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC. Clarke MT, et al. Among authors: mackenzie tc. Mol Ther. 2024 Apr 3;32(4):935-951. doi: 10.1016/j.ymthe.2024.02.004. Epub 2024 Feb 6. Mol Ther. 2024. PMID: 38327047 Free PMC article.
Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.
Herzeg A, Borges B, Lianoglou BR, Gonzalez-Velez J, Canepa E, Munar D, Young SP, Bali D, Gelb MH, Chakraborty P, Kishnani PS, Harmatz P, Cohen JL, MacKenzie TC. Herzeg A, et al. Among authors: mackenzie tc. Prenat Diagn. 2023 Dec;43(13):1638-1649. doi: 10.1002/pd.6460. Epub 2023 Nov 13. Prenat Diagn. 2023. PMID: 37955580 Free PMC article. Review.
95 results