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Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing.
Knipping F, Newby GA, Eide CR, McElroy AN, Nielsen SC, Smith K, Fang Y, Cornu TI, Costa C, Gutierrez-Guerrero A, Bingea SP, Feser CJ, Steinbeck B, Hippen KL, Blazar BR, McCaffrey A, Mussolino C, Verhoeyen E, Tolar J, Liu DR, Osborn MJ. Knipping F, et al. Among authors: mccaffrey a. Mol Ther. 2022 Jan 5;30(1):130-144. doi: 10.1016/j.ymthe.2021.10.026. Epub 2021 Nov 2. Mol Ther. 2022. PMID: 34737067 Free PMC article.
Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, Sheppard-Tillman H, Porter SN, Yao Y, Mayberry K, Everette KA, Jang Y, Podracky CJ, Thaman E, Lechauve C, Sharma A, Henderson JM, Richter MF, Zhao KT, Miller SM, Wang T, Koblan LW, McCaffrey AP, Tisdale JF, Kalfa TA, Pruett-Miller SM, Tsai SQ, Weiss MJ, Liu DR. Newby GA, et al. Among authors: mccaffrey ap. Nature. 2021 Jul;595(7866):295-302. doi: 10.1038/s41586-021-03609-w. Epub 2021 Jun 2. Nature. 2021. PMID: 34079130 Free PMC article.
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification.
Vaidyanathan S, Azizian KT, Haque AKMA, Henderson JM, Hendel A, Shore S, Antony JS, Hogrefe RI, Kormann MSD, Porteus MH, McCaffrey AP. Vaidyanathan S, et al. Among authors: mccaffrey ap. Mol Ther Nucleic Acids. 2018 Sep 7;12:530-542. doi: 10.1016/j.omtn.2018.06.010. Epub 2018 Jun 30. Mol Ther Nucleic Acids. 2018. PMID: 30195789 Free PMC article.
A story of mice and men.
McCaffrey AP, Kay MA. McCaffrey AP, et al. Gene Ther. 2002 Dec;9(23):1563. doi: 10.1038/sj.gt.3301890. Gene Ther. 2002. PMID: 12424608 No abstract available.
RNA interference in adult mice.
McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. McCaffrey AP, et al. Nature. 2002 Jul 4;418(6893):38-9. doi: 10.1038/418038a. Nature. 2002. PMID: 12097900
55 results