CRISPR/Cas9 therapeutics: progress and prospects

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and effici...

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Published inSignal transduction and targeted therapy Vol. 8; no. 1; pp. 36 - 23
Main Authors Li, Tianxiang, Yang, Yanyan, Qi, Hongzhao, Cui, Weigang, Zhang, Lin, Fu, Xiuxiu, He, Xiangqin, Liu, Meixin, Li, Pei-feng, Yu, Tao
Format Journal Article
LanguageEnglish
Published London Nature Publishing Group UK 16.01.2023
Nature Publishing Group
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Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from disease onset are expected to serve as identifying factors for targeted delivery or targets for gene editing. Diseases are both varied and complex, and the choice of appropriate gene-editing methods and delivery vectors for different diseases is important. Meanwhile, there are still many potential challenges identified when targeting delivery of CRISPR/Cas9 technology for disease treatment. This paper reviews the current developments in three aspects, namely, gene-editing type, delivery vector, and disease characteristics. Additionally, this paper summarizes successful examples of clinical trials and finally describes possible problems associated with current CRISPR applications.
AbstractList Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from disease onset are expected to serve as identifying factors for targeted delivery or targets for gene editing. Diseases are both varied and complex, and the choice of appropriate gene-editing methods and delivery vectors for different diseases is important. Meanwhile, there are still many potential challenges identified when targeting delivery of CRISPR/Cas9 technology for disease treatment. This paper reviews the current developments in three aspects, namely, gene-editing type, delivery vector, and disease characteristics. Additionally, this paper summarizes successful examples of clinical trials and finally describes possible problems associated with current CRISPR applications.
Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from disease onset are expected to serve as identifying factors for targeted delivery or targets for gene editing. Diseases are both varied and complex, and the choice of appropriate gene-editing methods and delivery vectors for different diseases is important. Meanwhile, there are still many potential challenges identified when targeting delivery of CRISPR/Cas9 technology for disease treatment. This paper reviews the current developments in three aspects, namely, gene-editing type, delivery vector, and disease characteristics. Additionally, this paper summarizes successful examples of clinical trials and finally describes possible problems associated with current CRISPR applications.
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from disease onset are expected to serve as identifying factors for targeted delivery or targets for gene editing. Diseases are both varied and complex, and the choice of appropriate gene-editing methods and delivery vectors for different diseases is important. Meanwhile, there are still many potential challenges identified when targeting delivery of CRISPR/Cas9 technology for disease treatment. This paper reviews the current developments in three aspects, namely, gene-editing type, delivery vector, and disease characteristics. Additionally, this paper summarizes successful examples of clinical trials and finally describes possible problems associated with current CRISPR applications.Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from disease onset are expected to serve as identifying factors for targeted delivery or targets for gene editing. Diseases are both varied and complex, and the choice of appropriate gene-editing methods and delivery vectors for different diseases is important. Meanwhile, there are still many potential challenges identified when targeting delivery of CRISPR/Cas9 technology for disease treatment. This paper reviews the current developments in three aspects, namely, gene-editing type, delivery vector, and disease characteristics. Additionally, this paper summarizes successful examples of clinical trials and finally describes possible problems associated with current CRISPR applications.
ArticleNumber 36
Author Li, Pei-feng
Yang, Yanyan
Liu, Meixin
He, Xiangqin
Cui, Weigang
Qi, Hongzhao
Li, Tianxiang
Zhang, Lin
Fu, Xiuxiu
Yu, Tao
Author_xml – sequence: 1
  givenname: Tianxiang
  surname: Li
  fullname: Li, Tianxiang
  organization: Institute for Translational Medicine, The Affiliated Hospital of Qingdao University
– sequence: 2
  givenname: Yanyan
  surname: Yang
  fullname: Yang, Yanyan
  organization: Department of Immunology, School of Basic Medicine, Qingdao University
– sequence: 3
  givenname: Hongzhao
  surname: Qi
  fullname: Qi, Hongzhao
  organization: Institute for Translational Medicine, The Affiliated Hospital of Qingdao University
– sequence: 4
  givenname: Weigang
  surname: Cui
  fullname: Cui, Weigang
  organization: Department of Cardiology, People’s Hospital of Rizhao
– sequence: 5
  givenname: Lin
  surname: Zhang
  fullname: Zhang, Lin
  organization: Department of Microbiology, Linyi Center for Disease Control and Prevention
– sequence: 6
  givenname: Xiuxiu
  surname: Fu
  fullname: Fu, Xiuxiu
  organization: Department of Cardiac Ultrasound, The Affiliated Hospital of Qingdao University
– sequence: 7
  givenname: Xiangqin
  surname: He
  fullname: He, Xiangqin
  organization: Department of Cardiac Ultrasound, The Affiliated Hospital of Qingdao University
– sequence: 8
  givenname: Meixin
  surname: Liu
  fullname: Liu, Meixin
  organization: Institute for Translational Medicine, The Affiliated Hospital of Qingdao University
– sequence: 9
  givenname: Pei-feng
  orcidid: 0000-0002-0969-9407
  surname: Li
  fullname: Li, Pei-feng
  email: peifli@qdu.edu.cn
  organization: Institute for Translational Medicine, The Affiliated Hospital of Qingdao University
– sequence: 10
  givenname: Tao
  surname: Yu
  fullname: Yu, Tao
  email: yutao0112@qdu.edu.cn
  organization: Institute for Translational Medicine, The Affiliated Hospital of Qingdao University, Department of Cardiac Ultrasound, The Affiliated Hospital of Qingdao University
BackLink https://www.ncbi.nlm.nih.gov/pubmed/36646687$$D View this record in MEDLINE/PubMed
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Yang, Chen, Shi (CR212) 2019; 31
Frangoul (CR74) 2021; 384
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Sousa, Coelho, Taipa (CR300) 2021; 97
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Nishida (CR167) 2016; 353
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CR113
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Chavez (CR145) 2015; 12
Fu (CR21) 2021; 49
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Shteinberg, Haq, Polineni, Davies (CR71) 2021; 397
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Chen (CR103) 2017; 550
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Landrum (CR23) 2016; 44
Burton (CR307) 2019; 19
Wang, Zhang, Gao (CR30) 2020; 181
Yang (CR234) 2019; 141
Liu (CR206) 2019; 6
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Maule, Ensinck, Bulcaen, Carlon (CR73) 2021; 182
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Chandrasegaran, Carroll (CR88) 2016; 428
Li (CR177) 2020; 48
Yüce, Filiztekin, Özkaya (CR289) 2021; 172
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Luther (CR121) 2018; 15
Barrangou (CR42) 2007; 315
Békési, Holub, Pálinkás, Vértessy (CR157) 2021; 22
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Kleinstiver (CR341) 2015; 523
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Zhang, Hu (CR263) 2015; 87
Schuster (CR175) 2019; 37
Luo (CR261) 2021; 28
Luk, Zhang (CR230) 2015; 220
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Tamay (CR210) 2019; 7
Marraffini, Sontheimer (CR9) 2010; 463
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Javaid, Pham, Choi (CR325) 2021; 22
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Qi (CR256) 2022; 11
Chen (CR12) 2019; 70
Liu (CR150) 2016; 167
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Huang (CR225) 2021; 17
Zhang, Zhang, Unver, Zhang (CR98) 2021; 29
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Schoger (CR276) 2020; 126
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Cox, Platt, Zhang (CR6) 2015; 21
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Zhao (CR181) 2018; 431
Wang (CR264) 2022; 20
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SSID ssj0001637754
ssib046561479
ssib044760960
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Snippet Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future...
Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the...
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StartPage 36
SubjectTerms 631/61/2300
692/4017
Cancer Research
Cell Biology
Clinical trials
CRISPR
CRISPR-Cas Systems - genetics
Disease
Gene Editing
Genetic Therapy - methods
Genome editing
Internal Medicine
Medicine
Medicine & Public Health
Oncology
Pathology
Review
Review Article
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Title CRISPR/Cas9 therapeutics: progress and prospects
URI https://link.springer.com/article/10.1038/s41392-023-01309-7
https://www.ncbi.nlm.nih.gov/pubmed/36646687
https://www.proquest.com/docview/2765886891
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https://pubmed.ncbi.nlm.nih.gov/PMC9841506
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Volume 8
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