First progeria monkey model generated using base editor

References

[1] Aguado J, Sola-Carvajal A, Cancila V, Revêchon G, Ong PF, JonesWeinert CW, Arzt EW, Lattanzi G, Dreesen O (2019) Inhibition of DNA damage response at telomeres improves the detrimental phenotypes of Hutchinson-Gilford Progeria syndrome. Nat Commun 10:2055

[2] Beyret E, Liao HK, Yamamoto M, Hernandez-Benitez R, Fu Y, Erikson G, Reddy P, Belmonte JC (2019) Single-dose CRISPR– Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome. Nat Med 119:1825

[3] Ceccaldi R, Rondinelli B, D’Andrea AD (2016) Repair pathway choices and consequences at the double-strand break. Trends Cell Biol 26:52–64

[4] Cox DB, Gootenberg JS, Abudayyeh OO, Franklin B, Kellner MJ, Joung J, Zhang F (2017) RNA editing with CRISPR-Cas13. Science 358:1019–1027

[5] Eriksson M, Brown WT, Gordon LB, Glynn MW, Singer J, Scott L, Erdos MR, Robbins CM, Moses TY, Berglund P (2003) Recurrent de novo point mutations in lamin A cause HutchinsonGilford progeria syndrome. Nature 423:293–298

[6] Fong LG (2006) A protein farnesyltransferase inhibitor ameliorates disease in a mouse model of progeria. Science 311:1621–1623

[7] Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI, Liu DR (2017) Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage. Nature 551:464–471

[8] Gorgoulis V, Adams PD, Alimonti A, Bennett DC, Bischof O, Bishop C, Campisi J, Collado M, Evangelou K, Ferbeyre G (2019) Cellular senescence: defining a path forward. Cell 179:813–827

[9] Komor AC, Kim YB, Packer MS, Zuris JA, Liu DR (2016) Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 533:420–424

[10] Liang P, Xie X, Zhi S, Sun H, Zhang X, Chen Y, Chen Y, Xiong Y, Ma W, Liu D (2019) Genome-wide profiling of adenine base editor specificity by EndoV-seq. Nat Commun 10:420

[11] Liu GH, Barkho BZ, Ruiz S, Diep D, Qu J, Yang SL, Panopoulos AD, Suzuki K, Kurian L, Walsh C (2011) Recapitulation of premature ageing with iPSCs from Hutchinson-Gilford progeria syndrome. Nature 472:221–225

[12] Nishida K, Arazoe T, Yachie N, Banno S, Kakimoto M, Tabata M, Mochizuki M, Miyabe A, Araki M, Hara KY, (2016) Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems. Science 353:aaf8729–aaf8729

[13] Niu Y, Shen B, Cui Y, Chen Y, Wang J, Wang L, Kang Y, Zhao X, Si W, Li W (2014) Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos. Cell 156:836–843

[14] Ocampo A, Reddy P, Martinez-Redondo P, Platero-Luengo A, Hatanaka F, Hishida T, Li M, Lam D, Kurita M, Beyret E (2016) In vivo amelioration of age-associated hallmarks by partial reprogramming. Cell 167:1719–1733.e12

[15] Osorio FG, Navarro CL, Cadiñanos J, López-Mejía IC, Quirós PM, Bartoli C, Rivera J, Tazi J, Guzmán G, Varela I, (2011) Splicing-directed therapy in a new mouse model of human accelerated aging. Science Translational Medicine 3:106ra107

[16] Ribes MP, Gualda EG, Doherty GJ, Espín DM (2019) Targeting senescent cells in translational medicine. EMBO Mol Med 11:6

[17] Santiago-Fernández O, Osorio FG, Quesada V, Rodríguez F, Basso S, Maeso D, Rolas L, Barkaway A, Nourshargh S, Folgueras AR (2019) Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome. Nat Med 25:423–426

[18] Suzuki K, Yamamoto M, Hernandez-Benitez R, Li Z, Wei C, Soligalla RD, Aizawa E, Hatanaka F, Kurita M, Reddy P (2019) Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction. Cell Res 21:121

[19] Wan H, Feng C, Teng F, Yang S, Hu B, Niu Y, Xiang AP, Fang W, Ji W, Li W (2014) One-step generation of p53 gene biallelic mutant Cynomolgus monkey via the CRISPR/Cas system. Cell Res 25:258–261

[20] Wu Z, Zhang W, Song M, Wang W, Wei G, Li W, Lei J, Huang Y, Sang Y, Chan P (2018) Differential stem cell aging kinetics in Hutchinson-Gilford progeria syndrome and Werner syndrome. Protein Cell 9:333–350

[21] Young SG, Yang SH, Davies BS, Jung HJ, Fong LG (2013) Targeting protein prenylation in progeria. Science Translational Medicine 5:171ps3–171ps3

[22] Zhang J, Lian Q, Zhu G, Zhou F, Sui L, Tan C, Mutalif RA, Navasankari R, Zhang Y, Tse HF (2011) A human iPSC model of hutchinson gilford progeria reveals vascular smooth muscle and mesenchymal stem cell defects. Stem Cell 8:31–45

[23] Zhang W, Wan H, Feng G, Qu J, Wang J, Jing Y, Ren R, Liu Z, Zhang L, Chen Z (2018) SIRT6 deficiency results in developmental retardation in cynomolgus monkeys. Nature 560:661–665

[24] Zhang X, Zhu B, Chen L, Xie L, Yu W, Wang Y, Li L, Yin S, Yang L, Hu H (2020) Dual base editor catalyzes both cytosine and adenine base conversions in human cells. Nat Biotechnol 533:420

[25] Zuo E, Cai YJ, Li K, Wei Y, Wang BA, Sun Y, Liu Z, Liu J, Hu X, Wei W (2017) One-step generation of complete gene knockout mice and monkeys by CRISPR/Cas9-mediated gene editing with multiple sgRNAs. Cell Res 27:933

RIGHTS & PERMISSIONS

2020 The Author(s)