Base Editing and Prime Editing: Potential Therapeutic Options for Rare and Common Diseases

Nguengang Wakap S, Lambert DM, Olry A, Rodwell C, Gueydan C, Lanneau V, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2019;28:165–73.

Article  PubMed  PubMed Central  Google Scholar 

Ferreira CR. The burden of rare diseases. Am J Med Genet A [Internet]. 2019 [cited 2022 Aug 25];179. https://pubmed.ncbi.nlm.nih.gov/30883013/.

Anguela XM, High KA. Entering the modern era of gene therapy. Annu Rev Med. 2019;70:273–88.

Article  CAS  PubMed  Google Scholar 

Newby GA, Liu DR. In vivo somatic cell base editing and prime editing. Mol Ther. 2021;29:3107–24.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021;6:1–24.

Google Scholar 

Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao G-P, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genets Metab. 2003;80:148–58.

Article  CAS  Google Scholar 

Gillmore JD, Maitland ML, Lebwohl D. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. Reply. N Engl J Med. 2021;385:1722–3.

Article  PubMed  Google Scholar 

Frangoul H, Altshuler D, Cappellini MD, Chen Y-S, Domm J, Eustace BK, et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med. 2021;384:252–60.

Article  CAS  PubMed  Google Scholar 

Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012;337:816–21.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Komor AC, Kim YB, Packer MS, Zuris JA, Liu DR. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature. 2016;533:420–4.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI, et al. Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage. Nature. 2017;551:464–71. https://doi.org/10.1038/nature24644.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, et al. Search-and-replace genome editing without double-strand breaks or donor DNA. Nature. 2019;576:149–57.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Mojica FJM, Rodriguez-Valera F. The discovery of CRISPR in archaea and bacteria. FEBS J. 2016;283:3162–9.

Article  CAS  PubMed  Google Scholar 

Jinek M, East A, Cheng A, Lin S, Ma E, Doudna J. RNA-programmed genome editing in human cells. Elife. 2013;2: e00471.

Article  PubMed  PubMed Central  Google Scholar 

Huang TP, Newby GA, Liu DR. Precision genome editing using cytosine and adenine base editors in mammalian cells. Nat Protoc. 2021;16:1089–128.

Article  CAS  PubMed  Google Scholar 

Miller JC, Tan S, Qiao G, Barlow KA, Wang J, Xia DF, et al. A TALE nuclease architecture for efficient genome editing. Nat Biotechnol. 2011;29:143–8.

Article  CAS  PubMed  Google Scholar 

Musunuru K. CRISPR and cardiovascular diseases. Cardiovasc Res. 2022;119:79–93.

Article  Google Scholar 

Walton RT, Christie KA, Whittaker MN, Kleinstiver BP. Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants. Science. 2020;368:290–6.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Thuronyi BW, Koblan LW, Levy JM, Yeh W-H, Zheng C, Newby GA, et al. Continuous evolution of base editors with expanded target compatibility and improved activity. Nat Biotechnol. 2019;37:1070–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Jiang W, Feng S, Huang S, Yu W, Li G, Yang G, et al. BE-PLUS: a new base editing tool with broadened editing window and enhanced fidelity. Cell Res. 2018;28:855–61.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kurt IC, Zhou R, Iyer S, Garcia SP, Miller BR, Langner LM, et al. CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells. Nat Biotechnol. 2021;39:41–6.

Article  CAS  PubMed  Google Scholar 

Koblan LW, Arbab M, Shen MW, Hussmann JA, Anzalone AV, Doman JL, et al. Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning. Nat Biotechnol. 2021;39:1414–25.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Zhao D, Li J, Li S, Xin X, Hu M, Price MA, et al. Glycosylase base editors enable C-to-A and C-to-G base changes. Nat Biotechnol. 2021;39:35–40.

Article  CAS  PubMed  Google Scholar 

Nelson JW, Randolph PB, Shen SP, Everette KA, Chen PJ, Anzalone AV, et al. Engineered pegRNAs improve prime editing efficiency. Nat Biotechnol. 2022;40:402–10.

Article  CAS  PubMed  Google Scholar 

Chen PJ, Hussmann JA, Yan J, Knipping F, Ravisankar P, Chen P-F, et al. Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. Cell. 2021;184:5635–52.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Levesque S, Mayorga D, Fiset J-P, Goupil C, Duringer A, Loiselle A, et al. Marker-free co-selection for successive rounds of prime editing in human cells. Nat Commun. 2022;13:5909.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Tao J, Wang Q, Mendez-Dorantes C, Burns KH, Chiarle R. Frequency and mechanisms of LINE-1 retrotransposon insertions at CRISPR/Cas9 sites. Nat Commun. 2022;13:3685.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Anzalone AV, Gao XD, Podracky CJ, Nelson AT, Koblan LW, Raguram A, et al. Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing. Nat Biotechnol. 2022;40:731–40.

Article  CAS  PubMed  Google Scholar 

Choi J, Chen W, Suiter CC, Lee C, Chardon FM, Yang W, et al. Precise genomic deletions using paired prime editing. Nat Biotechnol. 2022;40:218–26.

Article  CAS  PubMed  Google Scholar 

Zhuang Y, Liu J, Wu H, Zhu Q, Yan Y, Meng H, et al. Increasing the efficiency and precision of prime editing with guide RNA pairs. Nat Chem Biol. 2022;18:29–37.

Article  CAS  PubMed  Google Scholar 

Tao R, Wang Y, Jiao Y, Hu Y, Li L, Jiang L, et al. Bi-PE: bi-directional priming improves CRISPR/Cas9 prime editing in mammalian cells. Nucleic Acids Res. 2022;50:6423–34.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Jiang T, Zhang X-O, Weng Z, Xue W. Deletion and replacement of long genomic sequences using prime editing. Nat Biotechnol. 2022;40:227–34.

Article  CAS  PubMed  Google Scholar 

da Silva JF, Oliveira GP, Arasa-Verge EA, Kagiou C, Moretton A, Timelthaler G, et al. Prime editing efficiency and fidelity are enhanced in the absence of mismatch repair. Nat Commun. 2022;13:760.

Article  Google Scholar 

Kim HK, Yu G, Park J, Min S, Lee S, Yoon S, et al. Predicting the efficiency of prime editing guide RNAs in human cells. Nat Biotechnol. 2021;39:198–206.

Article  CAS  PubMed  Google Scholar 

Mathis N, Allam A, Kissling L, Marquart KF, Schmidheini L, Solari C, et al. Predicting prime editing efficiency and product purity by deep learning. Nat Biotechnol. 2023. https://doi.org/10.1038/s41587-022-01613-7.

Article  PubMed  Google Scholar 

Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong Z-M, et al. In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. Nature. 2021;589:608–14.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Olive M, Harten I, Mitchell R, Beers JK, Djabali K, Cao K, et al. Cardiovascular pathology in Hutchinson-Gilford progeria: correlation with the vascular pathology of aging. Arterioscler Thromb Vasc Biol. 2010. https://doi.org/10.1161/ATVBAHA.110.209460.

Article  PubMed  PubMed Central 

Comments (0)

No login
gif