Kathiresan S, Srivastava D. Genetics of human cardiovascular disease. Cell. 2012;148(6):1242–57.
Article CAS PubMed PubMed Central Google Scholar
Tada H, et al. Human genetics and its impact on cardiovascular disease. J Cardiol. 2022;79(2):233–9.
Rordorf R, et al. Real-world data of patients affected by advanced heart failure treated with implantable cardioverter defibrillator and left ventricular assist device: results of a multicenter observational study. Artif Organs. 2024;48(5):525–35.
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
de la Fuente-Nunez C, Lu TK. CRISPR-Cas9 technology: applications in genome engineering, development of sequence-specific antimicrobials, and future prospects. Integr Biol (Camb). 2017;9(2):109–22.
Hirakawa MP, et al. Gene editing and CRISPR in the clinic: current and future perspectives. Biosci Rep. 2020;40(4):BSR20200127.
Article CAS PubMed PubMed Central Google Scholar
Naeem M, et al. Latest developed strategies to minimize the off-target effects in CRISPR-cas-mediated genome editing. Cells. 2020;9(7):1608.
Article CAS PubMed PubMed Central Google Scholar
Bizy A, Klos M. Optimizing the use of iPSC-CMs for cardiac regeneration in animal models. Animals (Basel). 2020;10(9):1561.
Yamada Y, Sadahiro T, Ieda M. Development of direct cardiac reprogramming for clinical applications. J Mol Cell Cardiol. 2023;178:1–8.
Article CAS PubMed Google Scholar
Barrangou R, et al. CRISPR provides acquired resistance against viruses in prokaryotes. Science. 2007;315(5819):1709–12.
Article CAS PubMed Google Scholar
Guo T, et al. Harnessing accurate non-homologous end joining for efficient precise deletion in CRISPR/Cas9-mediated genome editing. Genome Biol. 2018;19:1–20.
Jang HK, et al. Current trends in gene recovery mediated by the CRISPR-Cas system. Exp Mol Med. 2020;52(7):1016–27.
Article CAS PubMed PubMed Central Google Scholar
Ran FA, et al. Genome engineering using the CRISPR-Cas9 system. Nat Protoc. 2013;8(11):2281–308.
Article CAS PubMed PubMed Central Google Scholar
Nakade S, et al. Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9. Nat Commun. 2014;5:5560.
Article CAS PubMed Google Scholar
Benitez EK, et al. Global and local manipulation of DNA repair mechanisms to alter site-specific gene editing outcomes in hematopoietic stem cells. Front Genome Edit. 2020;2:601541.
Sakuma T, et al. MMEJ-assisted gene knock-in using TALENs and CRISPR-Cas9 with the PITCh systems. Nat Protoc. 2016;11(1):118–33.
Article CAS PubMed Google Scholar
Komor AC, et al. 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, 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
Nishida K, et al. Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems. Science. 2016;353(6305):aaf8729.
Huang TP, Newby GA, Liu DR. Precision genome editing using cytosine and adenine base editors in mammalian cells. Nat Protoc. 2021;16(2):1089–128.
Article CAS PubMed Google Scholar
Komor AC, 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
Koblan LW, 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
Gaudelli NM, 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
Anzalone AV, 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
Chen PJ, Liu DR. Prime editing for precise and highly versatile genome manipulation. Nat Rev Genet. 2023;24(3):161–77.
Article CAS PubMed Google Scholar
Maggio I, et al. Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components. Gene Ther. 2020;27(5):209–25.
Article CAS PubMed PubMed Central Google Scholar
Liu P, et al. Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. Nat Commun. 2021;12(1):2121.
Article CAS PubMed PubMed Central Google Scholar
Chen PJ, et al. Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. Cell. 2021;184(22):5635-5652 e29.
Article CAS PubMed PubMed Central Google Scholar
Gao Z, et al. A truncated reverse transcriptase enhances prime editing by split AAV vectors. Mol Ther. 2022;30(9):2942–51.
Article CAS PubMed PubMed Central Google Scholar
Zheng C, et al. A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver. Mol Ther. 2022;30(3):1343–51.
Article CAS PubMed PubMed Central Google Scholar
Chen X, Gonçalves MAFV. Engineered viruses as genome editing devices. Mol Ther. 2016;24(3):447–57.
留言 (0)