Review
Medicine, Research & Experimental
Asma Ghaemi, Elnaz Bagheri, Khalil Abnous, Seyed Mohammad Taghdisi, Mohammad Ramezani, Mona Alibolandi
Summary: The prokaryotic CRISPR-Cas systems serve as a revolutionary genome editing tool for modifying, visualizing, and identifying DNA and RNA sequences in live cells of various species. Besides genome editing, the DNA-targeting modifications by CRISPR-Cas offer opportunities for diagnosis, therapy, and genetic disorders investigation. Preclinical studies and CRISPR-Cas9-based therapeutics may encounter challenges, but there is potential for its development in cancer therapy.
Review
Biochemistry & Molecular Biology
Si-Wei Wang, Chao Gao, Yi-Min Zheng, Li Yi, Jia-Cheng Lu, Xiao-Yong Huang, Jia-Bin Cai, Peng-Fei Zhang, Yue-Hong Cui, Ai-Wu Ke
Summary: The CRISPR/Cas9 gene editing technology has made significant progress in oncological research, providing a powerful tool for exploring the mechanism of cancer, establishing tumor models, and studying drug targets.
Review
Materials Science, Multidisciplinary
Shao Wei Hu, Tao Ding, Honghai Tang, Huiping Guo, Wenguo Cui, Yilai Shu
Summary: With the advancement of genome editing techniques, gene therapy is increasingly being used for the treatment of various human diseases. Nanomaterials have shown great potential in improving the efficiency and safety of gene editing in gene therapy. This review introduces three gene editing tools, with a particular focus on the development and optimization of the CRISPR system. It also discusses the recent application of nanobiomaterials in gene therapy, along with the prospects and remaining challenges. Lastly, future directions for gene editing tools, nanobiomaterial vectors, and gene therapy are presented.
Article
Biology
Ganna Reint, Zhuokun Li, Kornel Labun, Salla Keskitalo, Inkeri Soppa, Katariina Mamia, Eero Tolo, Monika Szymanska, Leonardo A. Meza-Zepeda, Susanne Lorenz, Artur Cieslar-Pobuda, Xian Hu, Diana L. Bordin, Judith Staerk, Eivind Valen, Bernhard Schmierer, Markku Varjosalo, Jussi Taipale, Emma Haapaniemi
Summary: Precision CRISPR gene editing can be improved by fusing DNA repair proteins with Cas9, with different fusion proteins showing varying effects based on cell type and genomic site. Optimization is necessary to account for the diverse factors contributing to locus-specific genome editing outcomes.
Review
Biotechnology & Applied Microbiology
Congting Guo, Xiaoteng Ma, Fei Gao, Yuxuan Guo
Summary: Gene editing involves precise changes to specific nucleic acid sequences. The CRISPR/Cas9 system has revolutionized gene editing, making it efficient, convenient, and programmable. However, off-target effects remain a major concern, leading to unexpected alterations in the genome. Various methods have been developed to detect and mitigate these off-target effects, advancing the precision of CRISPR/Cas9 derivatives. This review summarizes these technological advancements and discusses the challenges in managing off-target effects for future gene therapy.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2023)
Article
Multidisciplinary Sciences
J. Boutin, J. Rosier, D. Cappellen, F. Prat, J. Toutain, P. Pennamen, J. Bouron, C. Rooryck, J. P. Merlio, I. Lamrissi-Garcia, G. Cullot, S. Amintas, V. Guyonnet-Duperat, C. Ged, J. M. Blouin, E. Richard, S. Dabernat, F. Moreau-Gaudry, A. Bedel
Summary: The study reveals the genotoxicity caused by CRISPR-Cas9 leading to loss of heterozygosity changes, while also highlighting the potential safety concerns of this technology for gene therapy.
NATURE COMMUNICATIONS
(2021)
Review
Biochemistry & Molecular Biology
Magdalena Hryhorowicz, Daniel Lipinski, Joanna Zeyland
Summary: The bacteria-derived CRISPR/Cas system is a versatile tool for genome engineering, with wide applications in various fields. However, challenges like immunological reactions and off-target effects remain. This review discusses the current classification of CRISPR systems, new genome-editing technologies, recent applications, as well as limitations, ethical issues, and challenges associated with the CRISPR/Cas system.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Plant Sciences
Guo-Qing Song, Grace Urban, John T. Ryner, Gan-Yuan Zhong
Summary: This study evaluated chimeric editing in gene editing, using a transgenic tobacco line and a CRISPR-Cas9 editing vector. It was found that chimeric editing primarily occurred during or after cell regeneration. Further transformation was conducted to enhance editing efficiency. The findings provide valuable information for understanding gene editing in plant cells using constitutively expressed CRISPR-Cas9 and gRNAs.
Review
Cell Biology
Heba M. Mansour, Aiman S. El-Khatib
Summary: This article reviews the pathogenesis of Parkinson's disease (PD) and its association with CRISPR/Cas9 gene editing technology. It introduces the kinases encoding genes related to PD and targeted control by CRISPR/Cas9 technology. It also discusses the off-target effects of the CRISPR/Cas9 system and how to address them, as well as the challenges and future directions in kinase research for PD.
AGEING RESEARCH REVIEWS
(2023)
Article
Biochemical Research Methods
Stefan J. Tekel, Nicholas Brookhouser, Kylie Standage-Beier, Xiao Wang, David A. Brafman
Summary: The introduction of transient reporters of editing enrichment (TREE) has enabled highly efficient single-base editing of human cells using a transient episomal fluorescent reporter, allowing for rapid generation of clonal editing efficiencies exceeding 80% in biallelic or multiplexed edited isogenic human pluripotent stem cell lines within approximately 3-4 weeks.
Review
Biochemistry & Molecular Biology
Chin-Kai Chuang, Wei-Ming Lin
Summary: Cas9-sgRNA is a key tool for genome editing, with its simplicity, unique RNA-guided DNA recognition activity, and independent nuclease activities. Different variants of Cas9, in combination with sgRNA, can achieve various effects on target site breakage. Additionally, engineered enzyme activities can be introduced using Cas9 derivatives, and methods for transient expression and biosafety have been developed to address off-target effects.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Biochemistry & Molecular Biology
Chengpeng Wang, Yang Li, Na Wang, Qin Yu, Yonghong Li, Junping Gao, Xiaofeng Zhou, Nan Ma
Summary: We established a platform for CRISPR/Cas9-mediated gene editing in rose using suspension cells, and successfully edited a gene involved in the ethylene signaling pathway. Our optimized CRISPR/Cas9 system provides a powerful tool for functional genomics, molecular breeding, and synthetic biology in rose.
JOURNAL OF INTEGRATIVE PLANT BIOLOGY
(2023)
Review
Cell Biology
Manuel M. Vicente, Miguel Chaves-Ferreira, Joao M. P. Jorge, Joao T. Proenca, Vasco M. Barreto
Summary: The repurposing of the CRISPR/Cas bacterial defense system as molecular tools has revolutionized gene editing, and efforts have been made since 2013 to decrease the frequency of unwanted mutations during CRISPR-based gene editing.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Article
Plant Sciences
Jiarui Jiang, Haitao Huang, Qian Gao, Yong Li, Haiying Xiang, Wanli Zeng, Li Xu, Xin Liu, Jing Li, Qili Mi, Lele Deng, Wenwu Yang, Jianduo Zhang, Guangyu Yang, Xuemei Li
Summary: This study investigates the broad impact of DFR knockout on various developmental phases and organs of tobacco. DFR-KO decreases catabolism and photosynthetic light reactions in tobacco leaves during the flowering stage, while increasing carbon fixation and disease resistance pathways. However, the effects of DFR-KO on tobacco growth progressively decline with time. This research provides valuable insights and theoretical foundations for tobacco breeding and the development of gene-edited strains.
Review
Pharmacology & Pharmacy
Daniel Allen, Nechama Kalter, Michael Rosenberg, Ayal Hendel
Summary: Genome engineering through targeted nucleases, especially CRISPR-Cas9, has revolutionized gene therapy research and has the potential to treat blood and immune system diseases. CRISPR-Cas9 homology-directed repair (HDR) represents a promising method for site-specific gene insertion or correction. Other methods, like viral gene addition, gene knockout through non-homologous end joining (NHEJ), and base or prime editing, also show promise but have significant limitations for treating genetic immune or blood disorders. This review aims to highlight the transformative benefits of HDR-mediated gene therapy and propose solutions for current challenges, in order to advance HDR-based gene therapy in CD34(+) hematopoietic stem progenitor cells (HSPCs).