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Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia.
Arnaoutova I, Aratyn-Schaus Y, Zhang L, Packer MS, Chen HD, Lee C, Gautam S, Gregoire FM, Leboeuf D, Boule S, Fernandez TP, Huang V, Cheng LI, Lung G, Bannister B, Decker J, Leete T, Shuang LS, Bock C, Kothiyal P, Grayson P, Mok KW, Quinn JJ, Young L, Barrera L, Ciaramella G, Mansfield BC, Chou JY. Arnaoutova I, et al. Among authors: gregoire fm. Nat Commun. 2024 Nov 10;15(1):9729. doi: 10.1038/s41467-024-54108-1. Nat Commun. 2024. PMID: 39523369 Free PMC article.
Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency.
Packer MS, Chowdhary V, Lung G, Cheng LI, Aratyn-Schaus Y, Leboeuf D, Smith S, Shah A, Chen D, Zieger M, Cafferty BJ, Yan B, Ciaramella G, Gregoire FM, Mueller C. Packer MS, et al. Among authors: gregoire fm. Mol Ther. 2022 Apr 6;30(4):1396-1406. doi: 10.1016/j.ymthe.2022.01.040. Epub 2022 Feb 2. Mol Ther. 2022. PMID: 35121111 Free PMC article.
Cytosine base editing inhibits hepatitis B virus replication and reduces HBsAg expression in vitro and in vivo.
Smekalova EM, Martinez MG, Combe E, Kumar A, Dejene S, Leboeuf D, Chen CY, Dorkin JR, Shuang LS, Kieft S, Young L, Barrera LA, Packer MS, Ciaramella G, Testoni B, Gregoire F, Zoulim F. Smekalova EM, et al. Mol Ther Nucleic Acids. 2023 Dec 27;35(1):102112. doi: 10.1016/j.omtn.2023.102112. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2023. PMID: 38292874 Free PMC article.
Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing.
Zhang X, Zhao W, Nguyen GN, Zhang C, Zeng C, Yan J, Du S, Hou X, Li W, Jiang J, Deng B, McComb DW, Dorkin R, Shah A, Barrera L, Gregoire F, Singh M, Chen D, Sabatino DE, Dong Y. Zhang X, et al. Sci Adv. 2020 Aug 21;6(34):eabc2315. doi: 10.1126/sciadv.abc2315. Print 2020 Aug. Sci Adv. 2020. PMID: 32937374 Free PMC article.
Gene Editing Technologies to Target HBV cccDNA.
Martinez MG, Smekalova E, Combe E, Gregoire F, Zoulim F, Testoni B. Martinez MG, et al. Viruses. 2022 Nov 28;14(12):2654. doi: 10.3390/v14122654. Viruses. 2022. PMID: 36560658 Free PMC article. Review.
MBX-102/JNJ39659100, a novel peroxisome proliferator-activated receptor-ligand with weak transactivation activity retains antidiabetic properties in the absence of weight gain and edema.
Gregoire FM, Zhang F, Clarke HJ, Gustafson TA, Sears DD, Favelyukis S, Lenhard J, Rentzeperis D, Clemens LE, Mu Y, Lavan BE. Gregoire FM, et al. Mol Endocrinol. 2009 Jul;23(7):975-88. doi: 10.1210/me.2008-0473. Epub 2009 Apr 23. Mol Endocrinol. 2009. PMID: 19389808 Free PMC article.
29 results