引导编辑研究进展及其应用
Research progress and biotechnological applications of the prime editing
- 合成生物学 2024年5卷第1期 页码：1-15
- 作者机构：
清华大学自动化系，北京信息科学与技术国家研究中心，生物信息学研究部，生物信息学教育部重点实验室，合成与系统生物学研究中心，北京 100084
- 作者简介：
[ "许志锰（1995—），男，博士研究生。研究方向为合成生物学、基因编辑。 E-mail：xuzm18@mails.tsinghua.edu.cn" ]
[ "谢震（1976—），男，博士，副教授。研究方向为人工分子机器的设计与控制、合成生物学、肿瘤基因组学、肿瘤基因与细胞治疗。 E-mail：zhenxie@tsinghua.edu.cn" ]
- 基金信息：国家自然科学基金(32171413;61721003)
DOI：10.12211/2096-8280.2023-038
中图分类号： Q812收稿：2023-06-08，
修回：2023-09-08，
纸质出版：2024-02-29
- 稿件说明：
移动端阅览
许志锰， 谢震. 引导编辑研究进展及其应用［J］. 合成生物学， 2024， 5（1）： 1-15
XU Zhimeng， XIE Zhen. Research progress and biotechnological applications of the prime editing［J］. Synthetic Biology Journal， 2024， 5（1）： 1-15
许志锰， 谢震. 引导编辑研究进展及其应用［J］. 合成生物学， 2024， 5（1）： 1-15 DOI： 10.12211/2096-8280.2023-038.
XU Zhimeng， XIE Zhen. Research progress and biotechnological applications of the prime editing［J］. Synthetic Biology Journal， 2024， 5（1）： 1-15 DOI： 10.12211/2096-8280.2023-038.
摘要
引导编辑器（prime editor，PE）是继碱基编辑器（base editor，BE）之后新问世的基于CRISPR/Cas（clustered regularly interspaced short palindromic repeats/CRISPR-associated）系统的基因编辑工具，可以在不造成DNA双链断裂的情况下引入碱基替换、插入和删除。PE因其全面的编辑能力，问世即受到全球学者的广泛关注，然而PE表达盒编码较长（>6 kb）、编辑效率较低等问题也亟待研究人员解决。PE的研究方向与BE有许多相似之处，本文首先梳理了学界对PE本身编辑效率和安全性的探索；然后重点介绍了PE效应蛋白、pegRNA和其他细胞因子三个方面对PE的改进手段，以及为方便PE应用而开发的自动化设计工具；最后梳理了PE在动植物以及基因治疗中的应用。方兴未艾的PE领域尽管还难称完善，但在提高编辑效率和改进安全性等方面已取得了许多重要进展。鉴于Cas9、BE等基因编辑工具已广泛应用于遗传病疗法，PE走向遗传病治疗值得期待。
Abstract
Prime Editor (PE) is an innovative gene editing tool based on the Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein (CRISPR/Cas) system
which has revolutionized multiple fields
including genetics
medicine
and agriculture. Emerging as a successor to Base Editor (BE)
PE has gained worldwide attention due to its ability to introduce base substitutions
insertions
and deletions without causing double-strand DNA breaks
which significantly reduces the risk of off-target effect and unwanted genetic change. Notwithstanding its immense potential
researchers need to address PE's long encoding sequence and low editing efficiency for its maximal applications. Researchers have been working relentlessly to explore and enhance the editing efficiency and safety of PE by modifying its protein scaffold
optimizing the guide RNA design
and identifying cellular factors that influence its activity. Improved PE variants have been developed with enhanced accuracy and efficiency as well as decreased off-target effect when compared with their initial versions
demonstrating their potential in gene editing-related applications. Several strategies have been investigated to enhance PE performance
including: ① Modifying the structure of PE proteins to increase their efficiency
specificity
and binding affinity
thereby significantly improving their editing activity. ② Optimizing the design of pegRNAs
such as modifying the length
composition
or structure
that can boost PE's editing efficiency. ③ Identifying and manipulating cellular factors
such as proteins and RNAs
that bear functional relationships with the PE system
thus greatly enhancing its gene editing capabilities. ④ Developing automated design tools to facilitate the customization of the PE system for specific applications
vastly improving its practicality in research and clinical settings. Finally
this article summarizes the applications of PE in engineering animals and plants and developing gene therapy. Despite much room for further improvement in PE
significant advances have been made in improving its editing efficiency and safety. The rapid development of Cas9 and BE for treating genetic diseases stands as compelling testimony to the potential of PE in advancing gene editing technologies and applications. With continued research and development
PE holds great promise for improving human health and well-being.
2
关键词
Keywords
references
ANZALONE A V , KOBLAN L W , LIU D R . Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors [J ] . Nature Biotechnology , 2020 , 38 ( 7 ): 824 - 844 .
KOMOR A C , BADRAN A H , LIU D R . CRISPR-based technologies for the manipulation of eukaryotic genomes [J ] . Cell , 2017 , 168 ( 1/2 ): 20 - 36 .
WILLIAMS A B , SCHUMACHER B . p53 in the DNA-damage-repair process [J ] . Cold Spring Harbor Perspectives in Medicine , 2016 , 6 ( 5 ): a026070 .
HAAPANIEMI E , BOTLA S , PERSSON J , et al . CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response [J ] . Nature Medicine , 2018 , 24 ( 7 ): 927 - 930 .
KOMOR A C , KIM Y B , PACKER M S , et al . Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage [J ] . Nature , 2016 , 533 ( 7603 ): 420 - 424 .
GAUDELLI N M , KOMOR A C , REES H A , et al . Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage [J ] . Nature , 2017 , 551 ( 7681 ): 464 - 471 .
KURT I C , ZHOU R H , IYER S , et al . CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells [J ] . Nature Biotechnology , 2021 , 39 ( 1 ): 41 - 46 .
CHEN L , HONG M J , LUAN C M , et al . Adenine transversion editors enable precise, efficient A•T-to-C•G base editing in mammalian cells and embryos [J/OL ] . Nature Biotechnology , 2023 [ 2023-07-14 ] . https://www.nature.com/articles/s41587-023-01821-9 https://www.nature.com/articles/s41587-023-01821-9 .
TONG H W , LIU N N , WEI Y H , et al . Programmable deaminase-free base editors for G-to-Y conversion by engineered glycosylase [J ] . National Science Review , 2023 , 10 ( 8 ): nwad143 .
HAN D Y , XIAO Q Q , WANG Y F , et al . Development of miniature base editors using engineered IscB nickase [J ] . Nature Methods , 2023 , 20 ( 7 ): 1029 - 1036 .
ANZALONE A V , RANDOLPH P B , DAVIS J R , et al . Search-and-replace genome editing without double-strand breaks or donor DNA [J ] . Nature , 2019 , 576 ( 7785 ): 149 - 157 .
KIM H K , YU G , PARK J , et al . Predicting the efficiency of prime editing guide RNAs in human cells [J ] . Nature Biotechnology , 2021 , 39 ( 2 ): 198 - 206 .
LIN Q P , ZONG Y , XUE C X , et al . Prime genome editing in rice and wheat [J ] . Nature Biotechnology , 2020 , 38 ( 5 ): 582 - 585 .
LIN Q P , JIN S , ZONG Y , et al . High-efficiency prime editing with optimized, paired pegRNAs in plants [J ] . Nature Biotechnology , 2021 , 39 ( 8 ): 923 - 927 .
PONNIENSELVAN K , LIU P P , NYALILE T , et al . Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency [J ] . Nucleic Acids Research , 2023 , 51 ( 13 ): 6966 - 6980 .
KIM H K , LEE S , KIM Y , et al . High-throughput analysis of the activities of xCas9, Sp Cas9-NG and Sp Cas9 at matched and mismatched target sequences in human cells [J ] . Nature Biomedical Engineering , 2020 , 4 ( 1 ): 111 - 124 .
CHARI R , MALI P , MOOSBURNER M , et al . Unraveling CRISPR-Cas9 genome engineering parameters via a library-on-library approach [J ] . Nature Methods , 2015 , 12 ( 9 ): 823 - 826 .
KIM H K , SONG M , LEE J N , et al . In vivo high-throughput profiling of CRISPR-Cpf1 activity [J ] . Nature Methods , 2017 , 14 ( 2 ): 153 - 159 .
KIM H K , MIN S , SONG M , et al . Deep learning improves prediction of CRISPR-Cpf1 guide RNA activity [J ] . Nature Biotechnology , 2018 , 36 ( 3 ): 239 - 241 .
KIM H K , KIM Y , LEE S , et al . Sp Cas9 activity prediction by Deep Sp Cas9, a deep learning-based model with high generalization performance [J ] . Science Advances , 2019 , 5 ( 11 ): eaax9249 .
YU G , KIM H K , PARK J , et al . Prediction of efficiencies for diverse prime editing systems in multiple cell types [J ] . Cell , 2023 , 186 ( 10 ): 2256 - 2272.e23 .
MATHIS N , ALLAM A , KISSLING L , et al . Predicting prime editing efficiency and product purity by deep learning [J ] . Nature Biotechnology , 2023 , 41 ( 8 ): 1151 - 1159 .
KOEPPEL J , WELLER J , PEETS E M , et al . Prediction of prime editing insertion efficiencies using sequence features and DNA repair determinants [J ] . Nature Biotechnology , 2023 , 41 : 1446 - 1456 .
KIM D Y , MOON S B , KO J H , et al . Unbiased investigation of specificities of prime editing systems in human cells [J ] . Nucleic Acids Research , 2020 , 48 ( 18 ): 10576 - 10589 .
HABIB O , HABIB G , HWANG G H , et al . Comprehensive analysis of prime editing outcomes in human embryonic stem cells [J ] . Nucleic Acids Research , 2022 , 50 ( 2 ): 1187 - 1197 .
GAO R Z , FU Z C , LI X Y , et al . Genomic and transcriptomic analyses of prime editing guide RNA-independent off-target effects by prime editors [J ] . The CRISPR Journal , 2022 , 5 ( 2 ): 276 - 293 .
KWON J , KIM M , BAE S , et al . TAPE-seq is a cell-based method for predicting genome-wide off-target effects of prime editor [J ] . Nature Communications , 2022 , 13 : 7975 .
LIANG S Q , LIU P P , PONNIENSELVAN K , et al . Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag [J ] . Nature Methods , 2023 , 20 ( 6 ): 898 - 907 .
JIN S , LIN Q P , LUO Y F , et al . Genome-wide specificity of prime editors in plants [J ] . Nature Biotechnology , 2021 , 39 ( 10 ): 1292 - 1299 .
LIU P P , LIANG S Q , ZHENG C W , et al . Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice [J ] . Nature Communications , 2021 , 12 : 2121 .
CHEN P J , HUSSMANN J A , YAN J , et al . Enhanced prime editing systems by manipulating cellular determinants of editing outcomes [J ] . Cell , 2021 , 184 ( 22 ): 5635 - 5652.e29 .
SONG M , LIM J M , MIN S , et al . Generation of a more efficient prime editor 2 by addition of the Rad51 DNA-binding domain [J ] . Nature Communications , 2021 , 12 : 5617 .
PARK S J , JEONG T Y , SHIN S K , et al . Targeted mutagenesis in mouse cells and embryos using an enhanced prime editor [J ] . Genome Biology , 2021 , 22 ( 1 ): 170 .
VELIMIROVIC M , ZANETTI L C , SHEN M W , et al . Peptide fusion improves prime editing efficiency [J ] . Nature Communications , 2022 , 13 ( 1 ): 3512 .
YARNALL M T N , IOANNIDI E I , SCHMITT-ULMS C , et al . Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases [J ] . Nature Biotechnology , 2023 , 41 ( 4 ): 500 - 512 .
LIU Y , YANG G , HUANG S H , et al . Enhancing prime editing by Csy4-mediated processing of pegRNA [J ] . Cell Research , 2021 , 31 ( 10 ): 1134 - 1136 .
TAO R , WANG Y H , HU Y , et al . WT-PE: prime editing with nuclease wild-type Cas9 enables versatile large-scale genome editing [J ] . Signal Transduction and Targeted Therapy , 2022 , 7 : 108 .
PETERKA M , AKRAP N , LI S Y , et al . Harnessing DSB repair to promote efficient homology-dependent and-independent prime editing [J ] . Nature Communications , 2022 , 13 ( 1 ): 1240 .
LEE J , LIM K , KIM A , et al . Prime editing with genuine Cas9 nickases minimizes unwanted indels [J ] . Nature Communications , 2023 , 14 : 1786 .
MANOJ F , TAI L W , WANG K S M , et al . Targeted insertion of large genetic payloads using Cas directed LINE-1 reverse transcriptase [J ] . Scientific Reports , 2021 , 11 : 23625 .
ZONG Y , LIU Y J , XUE C X , et al . An engineered prime editor with enhanced editing efficiency in plants [J ] . Nature Biotechnology , 2022 , 40 ( 9 ): 1394 - 1402 .
XU W , YANG Y X , YANG B Y , et al . A design optimized prime editor with expanded scope and capability in plants [J ] . Nature Plants , 2022 , 8 ( 1 ): 45 - 52 .
ZHENG C W , LIANG S Q , LIU B , et al . A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver [J ] . Molecular Therapy , 2022 , 30 ( 3 ): 1343 - 1351 .
BÖCK D , ROTHGANGL T , VILLIGER L , et al . In vivo prime editing of a metabolic liver disease in mice [J ] . Science Translational Medicine , 2022 , 14 ( 636 ): eabl9238 .
GAO Z L , RAVENDRAN S , MIKKELSEN N S , et al . A truncated reverse transcriptase enhances prime editing by split AAV vectors [J ] . Molecular Therapy , 2022 , 30 ( 9 ): 2942 - 2951 .
LIU B , DONG X L , CHENG H Y , et al . A split prime editor with untethered reverse transcriptase and circular RNA template [J ] . Nature Biotechnology , 2022 , 40 ( 9 ): 1388 - 1393 .
KWEON J , YOON J K , JANG A H , et al . Engineered prime editors with PAM flexibility [J ] . Molecular Therapy , 2021 , 29 ( 6 ): 2001 - 2007 .
OH Y , LEE W J , HUR J K , et al . Expansion of the prime editing modality with Cas9 from Francisella novicida [J ] . Genome Biology , 2022 , 23 ( 1 ): 92 .
ZHANG X H , CHEN L , ZHU B Y , et al . Increasing the efficiency and targeting range of cytidine base editors through fusion of a single-stranded DNA-binding protein domain [J ] . Nature Cell Biology , 2020 , 22 ( 6 ): 740 - 750 .
CHEN R H , CAO Y , LIU Y J , et al . Enhancement of a prime editing system via optimal recruitment of the pioneer transcription factor P65 [J ] . Nature Communications , 2023 , 14 : 257 .
VELIMIROVIC M , ZANETTI L C , SHEN M W , et al . Peptide fusion improves prime editing efficiency [J ] . Nature Communications , 2022 , 13 ( 1 ): 3512 .
GRÜNEWALD J , MILLER B R , SZALAY R N , et al . Engineered CRISPR prime editors with compact, untethered reverse transcriptases [J ] . Nature Biotechnology , 2023 , 41 ( 3 ): 337 - 343 .
NELSON J W , RANDOLPH P B , SHEN S P , et al . Engineered pegRNAs improve prime editing efficiency [J ] . Nature Biotechnology , 2022 , 40 ( 3 ): 402 - 410 .
ZHANG G Q , LIU Y , HUANG S S , et al . Enhancement of prime editing via xrRNA motif-joined pegRNA [J ] . Nature Communications , 2022 , 13 : 1856 .
LI X Y , WANG X , SUN W J , et al . Enhancing prime editing efficiency by modified pegRNA with RNA G-quadruplexes [J ] . Journal of Molecular Cell Biology , 2022 , 14 ( 4 ): mjac022 .
LI X S , ZHOU L N , GAO B Q , et al . Highly efficient prime editing by introducing same-sense mutations in pegRNA or stabilizing its structure [J ] . Nature Communications , 2022 , 13 : 1669 .
RIESENBERG S , HELMBRECHT N , KANIS P , et al . Improved gRNA secondary structures allow editing of target sites resistant to CRISPR-Cas9 cleavage [J ] . Nature Communications , 2022 , 13 : 489 .
ZHUANG Y , LIU J L , WU H , et al . Increasing the efficiency and precision of prime editing with guide RNA pairs [J ] . Nature Chemical Biology , 2022 , 18 ( 1 ): 29 - 37 .
ANZALONE A V , GAO X D , PODRACKY C J , et al . Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing [J ] . Nature Biotechnology , 2022 , 40 ( 5 ): 731 - 740 .
CHOI J , CHEN W , SUITER C C , et al . Precise genomic deletions using paired prime editing [J ] . Nature Biotechnology , 2022 , 40 ( 2 ): 218 - 226 .
WANG J L , HE Z , WANG G Q , et al . Efficient targeted insertion of large DNA fragments without DNA donors [J ] . Nature Methods , 2022 , 19 ( 3 ): 331 - 340 .
JIANG T T , ZHANG X O , WENG Z P , et al . Deletion and replacement of long genomic sequences using prime editing [J ] . Nature Biotechnology , 2022 , 40 ( 2 ): 227 - 234 .
KWEON J , HWANG H Y , RYU H , et al . Targeted genomic translocations and inversions generated using a paired prime editing strategy [J ] . Molecular Therapy , 2023 , 31 ( 1 ): 249 - 259 .
TAO R , WANG Y H , JIAO Y G , et al . Bi-PE: bi-directional priming improves CRISPR/Cas9 prime editing in mammalian cells [J ] . Nucleic Acids Research , 2022 , 50 ( 11 ): 6423 - 6434 .
FERREIRA DA SILVA J , OLIVEIRA G P , ARASA-VERGE E A , et al . Prime editing efficiency and fidelity are enhanced in the absence of mismatch repair [J ] . Nature Communications , 2022 , 13 : 760 .
LIU N , ZHOU L F , LIN G F , et al . HDAC inhibitors improve CRISPR-Cas9 mediated prime editing and base editing [J ] . Molecular Therapy - Nucleic Acids , 2022 , 30 : 173 .
IHRY R J , WORRINGER K A , SALICK M R , et al . p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells [J ] . Nature Medicine , 2018 , 24 ( 7 ): 939 - 946 .
LI M , ZHONG A , WU Y J , et al . Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells [J ] . Nature Communications , 2022 , 13 : 6354 .
HUANG S S , ZHANG Z W , TAO W Y , et al . Broadening prime editing toolkits using RNA-Pol-Ⅱ-driven engineered pegRNA [J ] . Molecular Therapy , 2022 , 30 ( 9 ): 2923 - 2932 .
STANDAGE-BEIER K , TEKEL S J , BRAFMAN D A , et al . Prime editing guide RNA design automation using PINE-CONE [J ] . ACS Synthetic Biology , 2021 , 10 ( 2 ): 422 - 427 .
SIEGNER S M , KARASU M E , SCHRÖDER M S , et al . PnB Designer: a web application to design prime and base editor guide RNAs for animals and plants [J ] . BMC Bioinformatics , 2021 , 22 ( 1 ): 101 .
LI Y C , CHEN J J , TSAI S Q , et al . Easy-Prime: a machine learning-based prime editor design tool [J ] . Genome Biology , 2021 , 22 ( 1 ): 235 .
MORRIS J A , RAHMAN J A , GUO X Y , et al . Automated design of CRISPR prime editors for 56, 000 human pathogenic variants [J ] . iScience , 2021 , 24 ( 11 ): 103380 .
CHOW R D , CHEN J S , SHEN J , et al . A web tool for the design of prime-editing guide RNAs [J ] . Nature Biomedical Engineering , 2021 , 5 ( 2 ): 190 - 194 .
HSU J Y , GRÜNEWALD J , SZALAY R , et al . PrimeDesign software for rapid and simplified design of prime editing guide RNAs [J ] . Nature Communications , 2021 , 12 : 1034 .
ADIKUSUMA F , LUSHINGTON C , ARUDKUMAR J , et al . Optimized nickase- and nuclease-based prime editing in human and mouse cells [J ] . Nucleic Acids Research , 2021 , 49 ( 18 ): 10785 - 10795 .
ANDERSON M V , HALDRUP J , THOMSEN E A , et al . pegIT-a web-based design tool for prime editing [J ] . Nucleic Acids Research , 2021 , 49 ( W1 ): W505 - W509 .
HWANG G H , JEONG Y K , HABIB O , et al . PE-Designer and PE-Analyzer: web-based design and analysis tools for CRISPR prime editing [J ] . Nucleic Acids Research , 2021 , 49 ( W1 ): W499 - W504 .
LI H Q , BUSQUETS O , VERMA Y , et al . Highly efficient generation of isogenic pluripotent stem cell models using prime editing [J ] . eLife , 2022 , 11 : 79208 .
SÜRÜN D , SCHNEIDER A , MIRCETIC J , et al . Efficient generation and correction of mutations in human iPS cells utilizing mRNAs of CRISPR base editors and prime editors [J ] . Genes , 2020 , 11 ( 5 ): 511 .
PETRI K , ZHANG W T , MA J Y , et al . CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells [J ] . Nature Biotechnology , 2022 , 40 ( 2 ): 189 - 193 .
WOLFF J H , HALDRUP J , THOMSEN E A , et al . piggyPrime: high-efficacy prime editing in human cells using piggyBac-based DNA transposition [J ] . Frontiers in Genome Editing , 2021 , 3 : 786893 .
EGGENSCHWILER R , GSCHWENDTBERGER T , FELSKI C , et al . A selectable all-in-one CRISPR prime editing piggyBac transposon allows for highly efficient gene editing in human cell lines [J ] . Scientific Reports , 2021 , 11 : 22154 .
ZHI S Y , CHEN Y X , WU G L , et al . Dual-AAV delivering split prime editor system for in vivo genome editing [J ] . Molecular Therapy , 2022 , 30 ( 1 ): 283 - 294 .
SUN L W , LI J , XIAO X . Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization [J ] . Nature Medicine , 2000 , 6 ( 5 ): 599 - 602 .
JANG H , JO D H , CHO C S , et al . Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases [J ] . Nature Biomedical Engineering , 2022 , 6 ( 2 ): 181 - 194 .
DAVIS J R , BANSKOTA S , LEVY J M , et al . Efficient prime editing in mouse brain, liver and heart with dual AAVs [J/OL ] . Nature Biotechnology , 2023 [ 2023-07-10 ] . https://www.nature.com/articles/s41587-023-01758-z https://www.nature.com/articles/s41587-023-01758-z .
WANG Q , LIU J , JANSSEN J M , et al . Broadening the reach and investigating the potential of prime editors through fully viral gene-deleted adenoviral vector delivery [J ] . Nucleic Acids Research , 2021 , 49 ( 20 ): 11986 - 12001 .
AULICINO F , PELOSSE M , TOELZER C , et al . Highly efficient CRISPR-mediated large DNA docking and multiplexed prime editing using a single baculovirus [J ] . Nucleic Acids Research , 2022 , 50 ( 13 ): 7783 - 7799 .
LIU Y , LI X Y , HE S T , et al . Efficient generation of mouse models with the prime editing system [J ] . Cell Discovery , 2020 , 6 : 27 .
GAO P , LYU Q , GHANAM A R , et al . Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression [J ] . Genome Biology , 2021 , 22 ( 1 ): 83 .
LIN J X , LIU X C , LU Z Y , et al . Modeling a cataract disorder in mice with prime editing [J ] . Molecular Therapy Nucleic Acids , 2021 , 25 : 494 - 501 .
QIAN Y Q , ZHAO D , SUI T T , et al . Efficient and precise generation of Tay-Sachs disease model in rabbit by prime editing system [J ] . Cell Discovery , 2021 , 7 : 50 .
KIM D E , LEE J H , BIN JI K , et al . Prime editor-mediated correction of a pathogenic mutation in purebred dogs [J ] . Scientific Reports , 2022 , 12 : 12905 .
ATSUTA Y , SUZUKI K , IIKAWA H , et al . Prime editing in chicken fibroblasts and primordial germ cells [J ] . Development , Growth & Differentiation, 2022 , 64 ( 9 ): 548 - 557 .
BOSCH J A , BIRCHAK G , PERRIMON N . Precise genome engineering in Drosophila using prime editing [J ] . Proceedings of the National Academy of Sciences of the United States of America , 2021 , 118 ( 1 ): e2021996118 .
KIM Y , HONG S A , YU J , et al . Adenine base editing and prime editing of chemically derived hepatic progenitors rescue genetic liver disease [J ] . Cell Stem Cell , 2021 , 28 ( 9 ): 1614 - 1624.e5 .
CHEMELLO F , CHAI A C , LI H , et al . Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing [J ] . Science Advances , 2021 , 7 ( 18 ): eabg4910 .
HAPPI MBAKAM C , ROUSSEAU J , LU Y Y , et al . Prime editing optimized RTT permits the correction of the c. 8713C > T mutation in DMD gene [J ] . Molecular Therapy Nucleic Acids , 2022 , 30 : 272 - 285 .
LV X J , ZHENG Z , ZHI X , et al . Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor [J ] . Gene Therapy , 2023 , 30 ( 1/2 ): 160 - 166 .
HONG S A , KIM S E , LEE A Y , et al . Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa [J ] . Molecular Therapy , 2022 , 30 ( 8 ): 2664 - 2679 .
ZHOU M J , TANG S Q , DUAN N N , et al . Targeted-deletion of a tiny sequence via prime editing to restore SMN expression [J ] . International Journal of Molecular Sciences , 2022 , 23 ( 14 ): 7941 .
ZHANG H K , ZHOU Q , CHEN H Y , et al . Prime editor 3 mediated beta-thalassemia mutations of the HBB gene in human erythroid progenitor cells [J ] . International Journal of Molecular Sciences , 2022 , 23 ( 9 ): 5002 .
ZHANG H K , SUN R L , FEI J , et al . Correction of beta-thalassemia IVS-Ⅱ-654 mutation in a mouse model using prime editing [J ] . International Journal of Molecular Sciences , 2022 , 23 ( 11 ): 5948 .
ABUHAMAD A Y , MOHAMAD ZAMBERI N N , LING S E , et al . Reverting TP53 mutation in breast cancer cells: prime editing workflow and technical considerations [J ] . Cells , 2022 , 11 ( 10 ): 1612 .
TREMBLAY G , ROUSSEAU J , MBAKAM C H , et al . Insertion of the Icelandic mutation (A673T) by prime editing: a potential preventive treatment for familial and sporadic Alzheimer's disease [J ] . The CRISPR Journal , 2022 , 5 ( 1 ): 109 - 122 .
XU R F , LI J , LIU X S , et al . Development of plant prime-editing systems for precise genome editing [J ] . Plant Communications , 2020 , 1 ( 3 ): 100043 .
LI Z S , MA R , LIU D , et al . A straightforward plant prime editing system enabled highly efficient precise editing of rice Waxy gene [J ] . Plant Science , 2022 , 323 : 111400 .
JIN S , LIN Q P , GAO Q , et al . Optimized prime editing in monocot plants using PlantPegDesigner and engineered plant prime editors (ePPEs) [J ] . Nature Protocols , 2023 , 18 ( 3 ): 831 - 853 .
XU R F , LIU X S , LI J , et al . Identification of herbicide resistance OsACC1 mutations via in planta prime-editing-library screening in rice [J ] . Nature Plants , 2021 , 7 ( 7 ): 888 - 892 .
JIANG Y Y , CHAI Y P , LU M H , et al . Prime editing efficiently generates W542L and S621I double mutations in two ALS genes in maize [J ] . Genome Biology , 2020 , 21 ( 1 ): 257 .
QIAO D X , WANG J Y , LU M H , et al . Optimized prime editing efficiently generates heritable mutations in maize [J ] . Journal of Integrative Plant Biology , 2023 , 65 ( 4 ): 900 - 906 .
JIANG Z Z , ABDULLAH , ZHANG S , et al . Development and optimization of CRISPR prime editing system in photoautotrophic cells [J ] . Molecules , 2022 , 27 ( 6 ): 1758 .
LU Y M , TIAN Y F , SHEN R D , et al . Precise genome modification in tomato using an improved prime editing system [J ] . Plant Biotechnology Journal , 2021 , 19 ( 3 ): 415 - 417 .
PERROUD P F , GUYON-DEBAST A , VEILLET F , et al . Prime Editing in the model plant Physcomitrium patens and its potential in the tetraploid potato [J ] . Plant Science , 2022 , 316 : 111162 .
BISWAS S , BRIDGELAND A , IRUM S , et al . Optimization of prime editing in rice, peanut, chickpea, and cowpea protoplasts by restoration of GFP activity [J ] . International Journal of Molecular Sciences , 2022 , 23 ( 17 ): 9809 .
0
浏览量
2
下载量
0
CSCD
- 网络PDF下载
- WORD下载
- Meta-XML下载
- BibTeX下载
- Endnote下载
- RefMan 下载
- NoteExpress下载
- NoteFirst下载
关联资源
相关文章
相关作者
相关机构
- 地址：北京市东城区青年湖南街13号 邮编：100000
- 联系电话：010-64519339
- 技术支持由北京北大方正电子有限公司提供 京ICP备09064830号-19
京公网安备11010802024621 - 本系统建议在Chrome、 IE9+ 以上版本浏览器阅读本站内容，360浏览器请切换至极速模式
- Cookies帮助我们提供服务并提供个性化体验。使用本网站，即表示您同意我们使用Cookies