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A framework for individualized splice-switching oligonucleotide therapy.
Kim J, Woo S, de Gusmao CM, Zhao B, Chin DH, DiDonato RL, Nguyen MA, Nakayama T, Hu CA, Soucy A, Kuniholm A, Thornton JK, Riccardi O, Friedman DA, El Achkar CM, Dash Z, Cornelissen L, Donado C, Faour KNW, Bush LW, Suslovitch V, Lentucci C, Park PJ, Lee EA, Patterson A, Philippakis AA, Margus B, Berde CB, Yu TW. Kim J, et al. Among authors: nguyen ma. Nature. 2023 Jul;619(7971):828-836. doi: 10.1038/s41586-023-06277-0. Epub 2023 Jul 12. Nature. 2023. PMID: 37438524 Free PMC article.
Whole Exome Sequencing Is the Preferred Strategy to Identify the Genetic Defect in Patients With a Probable or Possible Mitochondrial Cause.
Theunissen TEJ, Nguyen M, Kamps R, Hendrickx AT, Sallevelt SCEH, Gottschalk RWH, Calis CM, Stassen APM, de Koning B, Mulder-Den Hartog ENM, Schoonderwoerd K, Fuchs SA, Hilhorst-Hofstee Y, de Visser M, Vanoevelen J, Szklarczyk R, Gerards M, de Coo IFM, Hellebrekers DMEI, Smeets HJM. Theunissen TEJ, et al. Front Genet. 2018 Oct 12;9:400. doi: 10.3389/fgene.2018.00400. eCollection 2018. Front Genet. 2018. PMID: 30369941 Free PMC article.
RNA therapeutics for disorders of excretory system.
Vu TD, Nguyen MA, Jurgoński A, Chu DT. Vu TD, et al. Among authors: nguyen ma. Prog Mol Biol Transl Sci. 2024;203:245-256. doi: 10.1016/bs.pmbts.2023.12.011. Epub 2024 Jan 25. Prog Mol Biol Transl Sci. 2024. PMID: 38360001
Non-Labeled, Stable Labeled, or Radiolabelled Approaches for Provision of Intravenous Pharmacokinetics in Humans: A Discussion Piece.
Young GC, Spracklin DK, James AD, Hvenegaard MG, Pedersen ML, Wagner DS, Georgi K, Schieferstein H, Bjornsdottir I, Romeo AA, Cassidy KC, Da-Violante G, Blech S, Schulz SI, Cuyckens F, Nguyen MA, Scarfe G. Young GC, et al. Among authors: nguyen ma. Clin Pharmacol Ther. 2024 May;115(5):931-938. doi: 10.1002/cpt.3121. Epub 2023 Dec 22. Clin Pharmacol Ther. 2024. PMID: 38018358 Review.
Phage for regenerative medicine and cosmetics.
Bui NL, Nguyen MA, Nguyen ML, Bui QC, Chu DT. Bui NL, et al. Among authors: nguyen ma. Prog Mol Biol Transl Sci. 2023;201:241-259. doi: 10.1016/bs.pmbts.2023.03.017. Epub 2023 Apr 6. Prog Mol Biol Transl Sci. 2023. PMID: 37770175
164 results