Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research | Military Medical Research

  • Doudna JA, Charpentier E. Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science. 2014;346(6213):1258096.

    Article 
    PubMed 

    Google Scholar
     

  • Doudna JA. The promise and challenge of therapeutic genome editing. Nature. 2020;578(7794):229–36.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Nambiar TS, Baudrier L, Billon P, Ciccia A. CRISPR-based genome editing through the lens of DNA repair. Mol Cell. 2022;82(2):348–88.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell. 2014;157(6):1262–78.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Komor AC, Badran AH, Liu DR. CRISPR-based technologies for the manipulation of eukaryotic genomes. Cell. 2017;169(3):559.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Knott GJ, Doudna JA. CRISPR-Cas guides the future of genetic engineering. Science. 2018;361(6405):866–9.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Li G, Li X, Zhuang S, Wang L, Zhu Y, Chen Y, et al. Gene editing and its applications in biomedicine. Sci China Life Sci. 2022;65(4):660–700.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Yang X. Applications of CRISPR-Cas9 mediated genome engineering. Mil Med Res. 2015;2:11.

    PubMed 
    PubMed Central 

    Google Scholar
     

  • Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339(6121):819–23.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Liu G, Lin Q, Jin S, Gao C. The CRISPR-Cas toolbox and gene editing technologies. Mol Cell. 2022;82(2):333–47.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Pickar-Oliver A, Gersbach CA. The next generation of CRISPR-Cas technologies and applications. Nat Rev Mol Cell Biol. 2019;20(8):490–507.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Porteus MH. A new class of medicines through DNA editing. N Engl J Med. 2019;380(10):947–59.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Yeh CD, Richardson CD, Corn JE. Advances in genome editing through control of DNA repair pathways. Nat Cell Biol. 2019;21(12):1468–78.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Clark JF, Dinsmore CJ, Soriano P. A most formidable arsenal: genetic technologies for building a better mouse. Genes Dev. 2020;34(19–20):1256–86.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Nishiga M, Liu C, Qi LS, Wu JC. The use of new CRISPR tools in cardiovascular research and medicine. Nat Rev Cardiol. 2022;19(8):505–21.

    Article 
    CAS 
    PubMed 

    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(7603):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(7681):464–71.

    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(7785):149–57.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Watkins WS, Hernandez EJ, Wesolowski S, Bisgrove BW, Sunderland RT, Lin E, et al. De novo and recessive forms of congenital heart disease have distinct genetic and phenotypic landscapes. Nat Commun. 2019;10(1):4722.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Jin SC, Homsy J, Zaidi S, Lu Q, Morton S, Depalma SR, et al. Contribution of rare inherited and de novo variants in 2,871 congenital heart disease probands. Nat Genet. 2017;49(11):1593–601.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Makarova KS, Wolf YI, Alkhnbashi OS, Costa F, Shah SA, Saunders SJ, et al. An updated evolutionary classification of CRISPR-Cas systems. Nat Rev Microbiol. 2015;13(11):722–36.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Dolan AE, Hou Z, Xiao Y, Gramelspacher MJ, Heo J, Howden SE, et al. Introducing a spectrum of long-range genomic deletions in human embryonic stem cells using type I CRISPR-Cas. Mol Cell. 2019;74(5):936-50.e5.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Morisaka H, Yoshimi K, Okuzaki Y, Gee P, Kunihiro Y, Sonpho E, et al. CRISPR-Cas3 induces broad and unidirectional genome editing in human cells. Nat Commun. 2019;10(1):5302.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Osakabe K, Wada N, Murakami E, Miyashita N, Osakabe Y. Genome editing in mammalian cells using the CRISPR type I-D nuclease. Nucleic Acids Res. 2021;49(11):6347–63.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Tan R, Krueger RK, Gramelspacher MJ, Zhou X, Xiao Y, Ke A, et al. Cas11 enables genome engineering in human cells with compact CRISPR-Cas3 systems. Mol Cell. 2022;82(4):852-67.e5.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Liu TY, Doudna JA. Chemistry of class 1 CRISPR-Cas effectors: binding, editing, and regulation. J Biol Chem. 2020;295(42):14473–87.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Altae-Tran H, Kannan S, Demircioglu FE, Oshiro R, Nety SP, Mckay LJ, et al. The widespread IS200/IS605 transposon family encodes diverse programmable RNA-guided endonucleases. Science. 2021;374(6563):57–65.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Karvelis T, Druteika G, Bigelyte G, Budre K, Zedaveinyte R, Silanskas A, et al. Transposon-associated TnpB is a programmable RNA-guided DNA endonuclease. Nature. 2021;599(7886):692–6.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Schuler G, Hu C, Ke A. Structural basis for RNA-guided DNA cleavage by IscB-ωRNA and mechanistic comparison with Cas9. Science. 2022;376(6600):1476–81.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Ran FA, Cong L, Yan WX, Scott DA, Gootenberg JS, Kriz AJ, et al. In vivo genome editing using Staphylococcus aureus Cas9. Nature. 2015;520(7546):186–91.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Hou Z, Zhang Y, Propson NE, Howden SE, Chu LF, Sontheimer EJ, et al. Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis. Proc Natl Acad Sci U S A. 2013;110(39):15644–9.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Kim E, Koo T, Park SW, Kim D, Kim K, Cho HY, et al. In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni. Nat Commun. 2017;8:14500.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Edraki A, Mir A, Ibraheim R, Gainetdinov I, Yoon Y, Song CQ, et al. A compact, high-accuracy Cas9 with a dinucleotide PAM for in vivo genome editing. Mol Cell. 2019;73(4):714-26 e4.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Liu JJ, Orlova N, Oakes BL, Ma E, Spinner HB, Baney KLM, et al. CasX enzymes comprise a distinct family of RNA-guided genome editors. Nature. 2019;566(7743):218–23.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Kim DY, Lee JM, Moon SB, Chin HJ, Park S, Lim Y, et al. Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus. Nat Biotechnol. 2022;40(1):94–102.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Wu Z, Zhang Y, Yu H, Pan D, Wang Y, Wang Y, et al. Programmed genome editing by a miniature CRISPR-Cas12f nuclease. Nat Chem Biol. 2021;17(11):1132–8.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Xu X, Chemparathy A, Zeng L, Kempton HR, Shang S, Nakamura M, et al. Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing. Mol Cell. 2021;81(20):4333-45.e4

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Pausch P, Al-Shayeb B, Bisom-Rapp E, Tsuchida CA, Li Z, Cress BF, et al. CRISPR-CasΦ from huge phages is a hypercompact genome editor. Science. 2020;369(6501):333–7.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Al-Shayeb B, Skopintsev P, Soczek KM, Stahl EC, Li Z, Groover E, et al. Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors. Cell. 2022;185(24):4574-86.e16.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Collias D, Beisel CL. CRISPR technologies and the search for the PAM-free nuclease. Nat Commun. 2021;12(1):555.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Miller SM, Wang T, Randolph PB, Arbab M, Shen MW, Huang TP, et al. Continuous evolution of SpCas9 variants compatible with non-G PAMs. Nat Biotechnol. 2020;38(4):471–81.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Walton RT, Christie KA, Whittaker MN, Kleinstiver BP. Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants. Science. 2020;368(6488):290–6.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Kleinstiver BP, Sousa AA, Walton RT, Tak YE, Hsu JY, Clement K, et al. Engineered CRISPR-Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing. Nat Biotechnol. 2019;37(3):276–82.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Tóth E, Varga É, Kulcsár PI, Kocsis-Jutka V, Krausz SL, Nyeste A, et al. Improved LbCas12a variants with altered PAM specificities further broaden the genome targeting range of Cas12a nucleases. Nucleic Acids Res. 2020;48(7):3722–33.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Chatterjee P, Jakimo N, Lee J, Amrani N, Rodriguez T, Koseki SRT, et al. An engineered ScCas9 with broad PAM range and high specificity and activity. Nat Biotechnol. 2020;38(10):1154–8.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Chatterjee P, Lee J, Nip L, Koseki SRT, Tysinger E, Sontheimer EJ, et al. A Cas9 with PAM recognition for adenine dinucleotides. Nat Commun. 2020;11(1):2474.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Ma D, Xu Z, Zhang Z, Chen X, Zeng X, Zhang Y, et al. Engineer chimeric Cas9 to expand PAM recognition based on evolutionary information. Nat Commun. 2019;10(1):560.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Liu RM, Liang LL, Freed E, Chang H, Oh E, Liu ZY, et al. Synthetic chimeric nucleases function for efficient genome editing. Nat Commun. 2019;10(1):5524.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Slaymaker IM, Gao L, Zetsche B, Scott DA, Yan WX, Zhang F. Rationally engineered Cas9 nucleases with improved specificity. Science. 2016;351(6268):84–8.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Kleinstiver BP, Pattanayak V, Prew MS, Tsai SQ, Nguyen NT, Zheng Z, et al. High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects. Nature. 2016;529(7587):490–5.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Chen JS, Dagdas YS, Kleinstiver BP, Welch MM, Sousa AA, Harrington LB, et al. Enhanced proofreading governs CRISPR-Cas9 targeting accuracy. Nature. 2017;550(7676):407–10.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Casini A, Olivieri M, Petris G, Montagna C, Reginato G, Maule G, et al. A highly specific SpCas9 variant is identified by in vivo screening in yeast. Nat Biotechnol. 2018;36(3):265–71.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, et al. A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells. Nat Med. 2018;24(8):1216–24.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Lee JK, Jeong E, Lee J, Jung M, Shin E, Kim YH, et al. Directed evolution of CRISPR-Cas9 to increase its specificity. Nat Commun. 2018;9(1):3048.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Tan Y, Chu AHY, Bao S, Hoang DA, Kebede FT, Xiong W, et al. Rationally engineered Staphylococcus aureus Cas9 nucleases with high genome-wide specificity. Proc Natl Acad Sci U S A. 2019;116(42):20969–76.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Xie H, Ge X, Yang F, Wang B, Li S, Duan J, et al. High-fidelity SaCas9 identified by directional screening in human cells. PLoS Biol. 2020;18(7):e3000747.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Bak SY, Jung Y, Park J, Sung K, Jang HK, Bae S, et al. Quantitative assessment of engineered Cas9 variants for target specificity enhancement by single-molecule reaction pathway analysis. Nucleic Acids Res. 2021;49(19):11312–22.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Kim N, Kim HK, Lee S, Seo JH, Choi JW, Park J, et al. Prediction of the sequence-specific cleavage activity of Cas9 variants. Nat Biotechnol. 2020;38(11):1328–36.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Schmid-Burgk JL, Gao L, Li D, Gardner Z, Strecker J, Lash B, et al. Highly parallel profiling of Cas9 variant specificity. Mol Cell. 2020;78(4):794-800.e8.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Bravo JPK, Liu MS, Hibshman GN, Dangerfield TL, Jung K, Mccool RS, et al. Structural basis for mismatch surveillance by CRISPR-Cas9. Nature. 2022;603(7900):343–7.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zhang L, Rube HT, Vakulskas CA, Behlke MA, Bussemaker HJ, Pufall MA. Systematic in vitro profiling of off-target affinity, cleavage and efficiency for CRISPR enzymes. Nucleic Acids Res. 2020;48(9):5037–53.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Tsuchida CA, Zhang S, Doost MS, Zhao Y, Wang J, O’brien E, et al. Chimeric CRISPR-CasX enzymes and guide RNAs for improved genome editing activity. Mol Cell. 2022;82(6):1199-209.e6.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Richardson CD, Ray GJ, Dewitt MA, Curie GL, Corn JE. Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA. Nat Biotechnol. 2016;34(3):339–44.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Maruyama T, Dougan SK, Truttmann MC, Bilate AM, Ingram JR, Ploegh HL. Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining. Nat Biotechnol. 2015;33(5):538–42.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Nishiyama J, Mikuni T, Yasuda R. Virus-mediated genome editing via homology-directed repair in mitotic and postmitotic cells in mammalian brain. Neuron. 2017;96(4):755-68.e5.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Ishizu T, Higo S, Masumura Y, Kohama Y, Shiba M, Higo T, et al. Targeted genome replacement via homology-directed repair in non-dividing cardiomyocytes. Sci Rep. 2017;7(1):9363.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zheng Y, Vandusen NJ, Butler CE, Ma Q, King JS, Pu WT. Efficient in vivo homology-directed repair within cardiomyocytes. Circulation. 2022;145(10):787–9.

    Article 
    PubMed 

    Google Scholar
     

  • Kosicki M, Tomberg K, Bradley A. Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements. Nat Biotechnol. 2018;36(8):765–71.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Cullot G, Boutin J, Toutain J, Prat F, Pennamen P, Rooryck C, et al. CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations. Nat Commun. 2019;10(1):1136.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Liu M, Zhang W, Xin C, Yin J, Shang Y, Ai C, et al. Global detection of DNA repair outcomes induced by CRISPR-Cas9. Nucleic Acids Res. 2021;49(15):8732–42.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Yu C, Liu Y, Ma T, Liu K, Xu S, Zhang Y, et al. Small molecules enhance CRISPR genome editing in pluripotent stem cells. Cell Stem Cell. 2015;16(2):142–7.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Riesenberg S, Maricic T. Targeting repair pathways with small molecules increases precise genome editing in pluripotent stem cells. Nat Commun. 2018;9(1):2164.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Genomes Project C, Auton A, Brooks LD, Durbin RM, Garrison EP, Kang HM, et al. A global reference for human genetic variation. Nature. 2015;526(7571):68–74.

    Article 

    Google Scholar
     

  • Eichler EE. Genetic variation, comparative genomics, and the diagnosis of disease. N Engl J Med. 2019;381(1):64–74.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Anzalone AV, Koblan LW, Liu DR. Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Nat Biotechnol. 2020;38(7):824–44.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Despres PC, Dube AK, Seki M, Yachie N, Landry CR. Perturbing proteomes at single residue resolution using base editing. Nat Commun. 2020;11(1):1871.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Hanna RE, Hegde M, Fagre CR, Deweirdt PC, Sangree AK, Szegletes Z, et al. Massively parallel assessment of human variants with base editor screens. Cell. 2021;184(4):1064-80.e20.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Cuella-Martin R, Hayward SB, Fan X, Chen X, Huang JW, Taglialatela A, et al. Functional interrogation of DNA damage response variants with base editing screens. Cell. 2021;184(4):1081-97.e19.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Komor AC, Zhao KT, Packer MS, Gaudelli NM, Waterbury AL, Koblan LW, et al. Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T: A base editors with higher efficiency and product purity. Sci Adv. 2017;3(8):eaao4774.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zafra MP, Schatoff EM, Katti A, Foronda M, Breinig M, Schweitzer AY, et al. Optimized base editors enable efficient editing in cells, organoids and mice. Nat Biotechnol. 2018;36(9):888–93.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Koblan LW, Doman JL, Wilson C, Levy JM, Tay T, Newby GA, et al. Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction. Nat Biotechnol. 2018;36(9):843–6.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Kim YB, Komor AC, Levy JM, Packer MS, Zhao KT, Liu DR. Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions. Nat Biotechnol. 2017;35(4):371–6.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Li X, Wang Y, Liu Y, Yang B, Wang X, Wei J, et al. Base editing with a Cpf1-cytidine deaminase fusion. Nat Biotechnol. 2018;36(4):324–7.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

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

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Huang TP, Zhao KT, Miller SM, Gaudelli NM, Oakes BL, Fellmann C, et al. Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors. Nat Biotechnol. 2019;37(6):626–31.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Richter MF, Zhao KT, Eton E, Lapinaite A, Newby GA, Thuronyi BW, et al. Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity. Nat Biotechnol. 2020;38(7):883–91.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Gaudelli NM, Lam DK, Rees HA, Sola-Esteves NM, Barrera LA, Born DA, et al. Directed evolution of adenine base editors with increased activity and therapeutic application. Nat Biotechnol. 2020;38(7):892–900.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Tan J, Zhang F, Karcher D, Bock R. Expanding the genome-targeting scope and the site selectivity of high-precision base editors. Nat Commun. 2020;11(1):629.

    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(1):41–6.

    Article 
    CAS 
    PubMed 

    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(1):35–40.

    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(11):1414–25.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Yuan T, Yan N, Fei T, Zheng J, Meng J, Li N, et al. Optimization of C-to-G base editors with sequence context preference predictable by machine learning methods. Nat Commun. 2021;12(1):4902.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zuo E, Sun Y, Wei W, Yuan T, Ying W, Sun H, et al. Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos. Science. 2019;364(6437):289–92.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Mcgrath E, Shin H, Zhang L, Phue JN, Wu WW, Shen RF, et al. Targeting specificity of APOBEC-based cytosine base editor in human iPSCs determined by whole genome sequencing. Nat Commun. 2019;10(1):5353.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Grunewald J, Zhou R, Garcia SP, Iyer S, Lareau CA, Aryee MJ, et al. Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors. Nature. 2019;569(7756):433–7.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zhou C, Sun Y, Yan R, Liu Y, Zuo E, Gu C, et al. Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis. Nature. 2019;571(7764):275–8.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Doman JL, Raguram A, Newby GA, Liu DR. Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors. Nat Biotechnol. 2020;38(5):620–8.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zuo E, Sun Y, Yuan T, He B, Zhou C, Ying W, et al. A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects. Nat Methods. 2020;17(6):600–4.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Wang L, Xue W, Zhang H, Gao R, Qiu H, Wei J, et al. Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations. Nat Cell Biol. 2021;23(5):552–63.

    Article 
    PubMed 

    Google Scholar
     

  • Chen PJ, Hussmann JA, Yan J, Knipping F, Ravisankar P, Chen PF, et al. Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. Cell. 2021;184(22):5635-52.e29.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    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(3):402–10.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Song M, Lim JM, Min S, Oh JS, Kim DY, Woo JS, et al. Generation of a more efficient prime editor 2 by addition of the Rad51 DNA-binding domain. Nat Commun. 2021;12(1):5617.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Park SJ, Jeong TY, Shin SK, Yoon DE, Lim SY, Kim SP, et al. Targeted mutagenesis in mouse cells and embryos using an enhanced prime editor. Genome Biol. 2021;22(1):170.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zong Y, Liu Y, Xue C, Li B, Li X, Wang Y, et al. An engineered prime editor with enhanced editing efficiency in plants. Nat Biotechnol. 2022;40(9):1394–402.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Jiang YY, Chai YP, Lu MH, Han XL, Lin Q, Zhang Y, et al. Prime editing efficiently generates W542L and S621I double mutations in two ALS genes in maize. Genome Biol. 2020;21(1):257.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    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(2):218–26.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Lin Q, Jin S, Zong Y, Yu H, Zhu Z, Liu G, et al. High-efficiency prime editing with optimized, paired pegRNAs in plants. Nat Biotechnol. 2021;39(8):923–7.

    Article 
    CAS 
    PubMed 

    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. 2021;40(5):731–40.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Jiang T, Zhang XO, Weng Z, Xue W. Deletion and replacement of long genomic sequences using prime editing. Nat Biotechnol. 2022;40(2):227–34.

    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(1):29–37.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Rybarski JR, Hu K, Hill AM, Wilke CO, Finkelstein IJ. Metagenomic discovery of CRISPR-associated transposons. Proc Natl Acad Sci U S A. 2021;118(49):e2112279118.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Klompe SE, Jaber N, Beh LY, Mohabir JT, Bernheim A, Sternberg SH. Evolutionary and mechanistic diversity of type I-F CRISPR-associated transposons. Mol Cell. 2022;82(3):616-28.e5.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Klompe SE, Vo PLH, Halpin-Healy TS, Sternberg SH. Transposon-encoded CRISPR-Cas systems direct RNA-guided DNA integration. Nature. 2019;571(7764):219–25.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Strecker J, Ladha A, Gardner Z, Schmid-Burgk JL, Makarova KS, Koonin EV, et al. RNA-guided DNA insertion with CRISPR-associated transposases. Science. 2019;365(6448):48–53.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Saito M, Ladha A, Strecker J, Faure G, Neumann E, Altae-Tran H, et al. Dual modes of CRISPR-associated transposon homing. Cell. 2021;184(9):2441-53.e18.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Vo PLH, Ronda C, Klompe SE, Chen EE, Acree C, Wang HH, et al. CRISPR RNA-guided integrases for high-efficiency, multiplexed bacterial genome engineering. Nat Biotechnol. 2021;39(4):480–9.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Pallarès-Masmitjà M, Ivančić D, Mir-Pedrol J, Jaraba-Wallace J, Tagliani T, Oliva B, et al. Find and cut-and-transfer (FiCAT) mammalian genome engineering. Nat Commun. 2021;12(1):7071.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Raguram A, Banskota S, Liu DR. Therapeutic in vivo delivery of gene editing agents. Cell. 2022;185(15):2806–27.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Wang D, Zhang F, Gao G. CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors. Cell. 2020;181(1):136–50.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature. 2021;595(7866):295–302.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, et al. Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease. Sci Transl Med. 2021;13(598):eabf2444.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zhang J, Hu Y, Yang J, Li W, Zhang M, Wang Q, et al. Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL. Nature. 2022;609(7926):369–74.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med. 2021;385(6):493–502.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, et al. Current clinical applications of in vivo gene therapy with AAVs. Mol Ther. 2021;29(2):464–88.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Chandler RJ, Sands MS, Venditti CP. Recombinant adeno-associated viral integration and genotoxicity: insights from animal models. Hum Gene Ther. 2017;28(4):314–22.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Banskota S, Raguram A, Suh S, Du SW, Davis JR, Choi EH, et al. Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. Cell. 2022;185(2):250-65.e16.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Sago CD, Lokugamage MP, Paunovska K, Vanover DA, Monaco CM, Shah NN, et al. High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing. Proc Natl Acad Sci U S A. 2018;115(42):E9944–52.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Ni H, Hatit MZC, Zhao K, Loughrey D, Lokugamage MP, Peck HE, et al. Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo. Nat Commun. 2022;13(1):4766.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Sago CD, Lokugamage MP, Loughrey D, Lindsay KE, Hincapie R, Krupczak BR, et al. Augmented lipid-nanoparticle-mediated in vivo genome editing in the lungs and spleen by disrupting Cas9 activity in the liver. Nat Biomed Eng. 2022;6(2):157–67.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Lyu P, Wang L, Lu B. Virus-like particle mediated CRISPR/Cas9 delivery for efficient and safe genome editing. Life (Basel). 2020;10(12):366.

    CAS 
    PubMed 

    Google Scholar
     

  • Lu Z, Yao X, Lyu P, Yadav M, Yoo K, Atala A, et al. Lentiviral capsid-mediated streptococcus pyogenes Cas9 ribonucleoprotein delivery for efficient and safe multiplex genome editing. CRISPR J. 2021;4(6):914–28.

    CAS 
    PubMed 

    Google Scholar
     

  • Hamilton JR, Tsuchida CA, Nguyen DN, Shy BR, Mcgarrigle ER, Sandoval Espinoza CR, et al. Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering. Cell Rep. 2021;35(9):109207.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Lyu P, Lu Z, Cho SI, Yadav M, Yoo KW, Atala A, et al. Adenine base editor ribonucleoproteins delivered by lentivirus-like particles show high on-target base editing and undetectable RNA off-target activities. CRISPR J. 2021;4(1):69–81.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Segel M, Lash B, Song J, Ladha A, Liu CC, Jin X, et al. Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery. Science. 2021;373(6557):882–9.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Roth GA, Mensah GA, Johnson CO, Addolorato G, Ammirati E, Baddour LM, et al. Global burden of cardiovascular diseases and risk factors, 1990–2019: update from the GBD 2019 study. J Am Coll Cardiol. 2020;76(25):2982–3021.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Leopold JA, Loscalzo J. Emerging role of precision medicine in cardiovascular disease. Circ Res. 2018;122(9):1302–15.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Madsen A, Höppner G, Krause J, Hirt MN, Laufer SD, Schweizer M, et al. An important role for DNMT3A-mediated DNA methylation in cardiomyocyte metabolism and contractility. Circulation. 2020;142(16):1562–78.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Bengel P, Dybkova N, Tirilomis P, Ahmad S, Hartmann N, Mohamed BA, et al. Detrimental proarrhythmogenic interaction of Ca2+/calmodulin-dependent protein kinase II and NaV1.8 in heart failure. Nat Commun. 2021;12(1):6586.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Levitas A, Muhammad E, Zhang Y, Perea Gil I, Serrano R, Diaz N, et al. A novel recessive mutation in SPEG causes early onset dilated cardiomyopathy. PLoS Genet. 2020;16(9):e1009000.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Fomin A, Gärtner A, Cyganek L, Tiburcy M, Tuleta I, Wellers L, et al. Truncated titin proteins and titin haploinsufficiency are targets for functional recovery in human cardiomyopathy due to TTN mutations. Sci Transl Med. 2021;13(618):eabd3079.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Ma S, Jiang W, Liu X, Lu WJ, Qi T, Wei J, et al. Efficient correction of a hypertrophic cardiomyopathy mutation by ABEmax-NG. Circ Res. 2021;129(10):895–908.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Huang JY, Kan SH, Sandfeld EK, Dalton ND, Rangel AD, Chan Y, et al. CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness. Sci Rep. 2020;10(1):10321.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Ercu M, Markó L, Schächterle C, Tsvetkov D, Cui Y, Maghsodi S, et al. Phosphodiesterase 3A and arterial hypertension. Circulation. 2020;142(2):133–49.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Min YL, Li H, Rodriguez-Caycedo C, Mireault AA, Huang J, Shelton JM, et al. CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells. Sci Adv. 2019;5(3):eaav4324.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Magdy T, Jouni M, Kuo HH, Weddle CJ, Lyra-Leite D, Fonoudi H, et al. Identification of drug transporter genomic variants and inhibitors that protect against doxorubicin-induced cardiotoxicity. Circulation. 2022;145(4):279–94.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Chemello F, Chai AC, Li H, Rodriguez-Caycedo C, Sanchez-Ortiz E, Atmanli A, et al. Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing. Sci Adv. 2021;7(18):eabg4910.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Johansen AK, Molenaar B, Versteeg D, Leitoguinho AR, Demkes C, Spanjaard B, et al. Postnatal cardiac gene editing using CRISPR/Cas9 with AAV9-mediated delivery of short guide RNAs results in mosaic gene disruption. Circ Res. 2017;121(10):1168–81.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Song X, Cui Y, Wang Y, Zhang Y, He Q, Yu Z, et al. Genome editing with AAV-BR1-CRISPR in postnatal mouse brain endothelial cells. Int J Biol Sci. 2022;18(2):652–60.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Xu L, Lau YS, Gao Y, Li H, Han R. Life-long AAV-mediated CRISPR genome editing in dystrophic heart improves cardiomyopathy without causing serious lesions in mdx mice. Mol Ther. 2019;27(8):1407–14.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Nelson CE, Wu Y, Gemberling MP, Oliver ML, Waller MA, Bohning JD, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nat Med. 2019;25(3):427–32.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Liu N, Olson EN. CRISPR modeling and correction of cardiovascular disease. Circ Res. 2022;130(12):1827–50.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Heimlich JB, Bick AG. Somatic mutations in cardiovascular disease. Circ Res. 2022;130(1):149–61.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Strong A, Musunuru K. Genome editing in cardiovascular diseases. Nat Rev Cardiol. 2017;14(1):11–20.

    Article 
    CAS 

    Google Scholar
     

  • Belbachir N, Portero V, Al Sayed ZR, Gourraud JB, Dilasser F, Jesel L, et al. RRAD mutation causes electrical and cytoskeletal defects in cardiomyocytes derived from a familial case of Brugada syndrome. Eur Heart J. 2019;40(37):3081–94.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zhang XH, Wei H, Xia Y, Morad M. Calcium signaling consequences of RyR2 mutations associated with CPVT1 introduced via CRISPR/Cas9 gene editing in human-induced pluripotent stem cell-derived cardiomyocytes: comparison of RyR2-R420Q, F2483I, and Q4201R. Heart Rhythm. 2021;18(2):250–60.

    Article 
    PubMed 

    Google Scholar
     

  • Pettinato AM, Ladha FA, Mellert DJ, Legere N, Cohn R, Romano R, et al. Development of a cardiac sarcomere functional genomics platform to enable scalable interrogation of uhman TNNT2 variants. Circulation. 2020;142(23):2262–75.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Guo F, Sun Y, Wang X, Wang H, Wang J, Gong T, et al. Patient-specific and gene-corrected induced pluripotent stem cell-derived cardiomyocytes elucidate single-cell phenotype of short QT syndrome. Circ Res. 2019;124(1):66–78.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Dotzler SM, Kim CSJ, Genfrom WAC, Zhou W, Ye D, Bos JM, et al. Suppression-replacement KCNQ1 gene therapy for type 1 long QT syndrome. Circulation. 2021;143(14):1411–25.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Lambert M, Capuano V, Boet A, Tesson L, Bertero T, Nakhleh MK, et al. Characterization of Kcnk3-mutated rat, a novel model of pulmonary hypertension. Circ Res. 2019;125(7):678–95.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Li J, Wang K, Zhang Y, Qi T, Yuan J, Zhang L, et al. Therapeutic exon skipping through a CRISPR-guided cytidine deaminase rescues dystrophic cardiomyopathy in vivo. Circulation. 2021;144(22):1760–76.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Li W, Tran V, Shaked I, Xue B, Moore T, Lightle R, et al. Abortive intussusceptive angiogenesis causes multi-cavernous vascular malformations. Elife. 2021;10:e62155.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Carroll KJ, Makarewich CA, Mcanally J, Anderson DM, Zentilin L, Liu N, et al. A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9. Proc Natl Acad Sci U S A. 2016;113(2):338–43.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Schoger E, Carroll KJ, Iyer LM, Mcanally JR, Tan W, Liu N, et al. CRISPR-mediated activation of endogenous gene expression in the postnatal heart. Circ Res. 2020;126(1):6–24.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Zhu W, Saw D, Weiss M, Sun Z, Wei M, Shaligram S, et al. Induction of brain arteriovenous malformation through CRISPR/Cas9-mediated somatic Alk1 gene mutations in adult mice. Transl Stroke Res. 2019;10(5):557–65.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Zhang X, Jin H, Huang X, Chaurasiya B, Dong D, Shanley TP, et al. Robust genome editing in adult vascular endothelium by nanoparticle delivery of CRISPR-Cas9 plasmid DNA. Cell Rep. 2022;38(1): 110196.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Liang P, Xu Y, Zhang X, Ding C, Huang R, Zhang Z, et al. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell. 2015;6(5):363–72.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Kang X, He W, Huang Y, Yu Q, Chen Y, Gao X, et al. Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing. J Assist Reprod Genet. 2016;33(5):581–8.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zuccaro MV, Xu J, Mitchell C, Marin D, Zimmerman R, Rana B, et al. Allele-specific chromosome removal after Cas9 cleavage in human embryos. Cell. 2020;183(6):1650-64.e15.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Turocy J, Adashi EY, Egli D. Heritable human genome editing: research progress, ethical considerations, and hurdles to clinical practice. Cell. 2021;184(6):1561–74.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Baylis F, Darnovsky M, Hasson K, Krahn TM. Human germ line and heritable genome editing: the global policy landscape. CRISPR J. 2020;3(5):365–77.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Min YL, Chemello F, Li H, Rodriguez-Caycedo C, Sanchez-Ortiz E, Mireault AA, et al. Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing. Mol Ther. 2020;28(9):2044–55.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Moretti A, Fonteyne L, Giesert F, Hoppmann P, Meier AB, Bozoglu T, et al. Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy. Nat Med. 2020;26(2):207–14.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Xu L, Zhang C, Li H, Wang P, Gao Y, Mokadam NA, et al. Efficient precise in vivo base editing in adult dystrophic mice. Nat Commun. 2021;12(1):3719.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Hanses U, Kleinsorge M, Roos L, Yigit G, Li Y, Barbarics B, et al. Intronic CRISPR repair in a preclinical model of noonan syndrome-associated cardiomyopathy. Circulation. 2020;142(11):1059–76.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Ma H, Marti-Gutierrez N, Park SW, Wu J, Lee Y, Suzuki K, et al. Correction of a pathogenic gene mutation in human embryos. Nature. 2017;548(7668):413–9.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Romano R, Ghahremani S, Zimmerman T, Legere N, Thakar K, Ladha FA, et al. Reading frame repair of TTN truncation variants restores titin quantity and functions. Circulation. 2022;145(3):194–205.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Nishiyama T, Zhang Y, Cui M, Li H, Sanchez-Ortiz E, Mcanally JR, et al. Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy. Sci Transl Med. 2022;14(672):eade1633.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Dave J, Raad N, Mittal N, Zhang L, Fargnoli A, Oh JG, et al. Gene editing reverses arrhythmia susceptibility in humanized PLN-R14del mice: modelling a European cardiomyopathy with global impact. Cardiovasc Res. 2022;118(15):3140–50.

    Article 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Ding Q, Strong A, Patel KM, Ng SL, Gosis BS, Regan SN, et al. Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing. Circ Res. 2014;115(5):488–92.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Musunuru K, Chadwick AC, Mizoguchi T, Garcia SP, Denizio JE, Reiss CW, et al. In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates. Nature. 2021;593(7859):429–34.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Rothgangl T, Dennis MK, Lin PJC, Oka R, Witzigmann D, Villiger L, et al. In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels. Nat Biotechnol. 2021;39(8):949–57.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987;51(6):919–28.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Hinson JT, Chopra A, Nafissi N, Polacheck WJ, Benson CC, Swist S, et al. Heart disease. Titin mutations in iPS cells define sarcomere insufficiency as a cause of dilated cardiomyopathy. Science. 2015;349(6251):982–6.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Chopra A, Kutys ML, Zhang K, Polacheck WJ, Sheng CC, Luu RJ, et al. Force generation via β-cardiac myosin, titin, and α-actinin drives cardiac sarcomere assembly from cell-matrix adhesions. Dev Cell. 2018;44(1):87-96.e5.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Zaunbrecher RJ, Abel AN, Beussman K, Leonard A, Von Frieling-Salewsky M, Fields PA, et al. Cronos titin is expressed in human cardiomyocytes and necessary for normal sarcomere function. Circulation. 2019;140(20):1647–60.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Xie C, Zhang YP, Song L, Luo J, Qi W, Hu J, et al. Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome. Cell Res. 2016;26(10):1099–111.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Pan X, Philippen L, Lahiri SK, Lee C, Park SH, Word TA, et al. In vivo Ryr2 editing corrects catecholaminergic polymorphic ventricular tachycardia. Circ Res. 2018;123(8):953–63.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Libby P. The changing landscape of atherosclerosis. Nature. 2021;592(7855):524–33.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Kannan S, Altae-Tran H, Jin X, Madigan VJ, Oshiro R, Makarova KS, et al. Compact RNA editors with small Cas13 proteins. Nat Biotechnol. 2022;40(2):194–7.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Kato K, Okazaki S, Schmitt-Ulms C, Jiang K, Zhou W, Ishikawa J, et al. RNA-triggered protein cleavage and cell growth arrest by the type III-E CRISPR nuclease-protease. Science. 2022;378(6622):882–9.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Strecker J, Demircioglu FE, Li D, Faure G, Wilkinson ME, Gootenberg JS, et al. RNA-activated protein cleavage with a CRISPR-associated endopeptidase. Science. 2022;378(6622):874–81.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, et al. CRISPR-Cas9 gene editing for sickle cell disease and beta-thalassemia. N Engl J Med. 2021;384(3):252–60.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Stadtmauer EA, Fraietta JA, Davis MM, Cohen AD, Weber KL, Lancaster E, et al. CRISPR-engineered T cells in patients with refractory cancer. Science. 2020;367(6481):eaba7365.

    Article 
    CAS 
    PubMed 

    Google Scholar
     

  • Grunwald HA, Gantz VM, Poplawski G, Xu XS, Bier E, Cooper KL. Super-Mendelian inheritance mediated by CRISPR-Cas9 in the female mouse germline. Nature. 2019;566(7742):105–9.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

  • Alanis-Lobato G, Zohren J, McCarthy A, Fogarty NME, Kubikova N, Hardman E, et al. Frequent loss of heterozygosity in CRISPR-Cas9-edited early human embryos. Proc Natl Acad Sci U S A. 2021;118(22):e2004832117.

    Article 
    CAS 
    PubMed 
    PubMed Central 

    Google Scholar
     

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