Review
Cell Biology
Narendranath Bhokisham, Ethan Laudermilch, Lindsay L. Traeger, Tonya D. Bonilla, Mercedes Ruiz-Estevez, Jordan R. Becker
Summary: CRISPR-Cas technology has revolutionized life science research and medicine by allowing manipulation of human DNA, potentially treating both genetic and complex diseases. This technology is being used in various clinical trials, and new CRISPR-Cas-based tools are further expanding its therapeutic potential. Additionally, the CRISPR-Cas system is also contributing to the understanding of human diseases through the creation of animal disease models for preclinical testing.
Review
Chemistry, Medicinal
Phei Er Saw, Guo-Hui Cui, Xiaoding Xu
Summary: This paper discusses the delivery pathways of the gene editing technology CRISPR/Cas system and its applications in clinical diseases. Nanoparticles have been proven to be a promising delivery system for CRISPR/Cas.
Review
Medicine, General & Internal
Songyang Zhang, Yidi Wang, Dezhi Mao, Yue Wang, Hong Zhang, Yihan Pan, Yuezeng Wang, Shuzhi Teng, Ping Huang
Summary: This review focuses on the trends in clinical studies involving CRISPR techniques, specifically in the treatment of cancer, hematological, endocrine, and immune system diseases, as well as in diagnostics. The scientific basis of CRISPR/Cas technologies is analyzed, and the challenges and recent advances in using CRISPR for disease therapies are discussed.
FRONTIERS IN MEDICINE
(2023)
Review
Plant Sciences
Irene Guzman-Benito, Natalia Patricia Achkar, Nicolas G. Bologna, Robertas Ursache
Summary: This paper reviews recent advances in CRISPR/Cas-mediated gene targeting in plants, discusses their potential applications, and suggests further improvements to achieve higher efficiency.
JOURNAL OF EXPERIMENTAL BOTANY
(2023)
Review
Biochemistry & Molecular Biology
Ali Dehshahri, Alessio Biagioni, Hadi Bayat, E. Hui Clarissa Lee, Mohammad Hashemabadi, Hojjat Samareh Fekri, Ali Zarrabi, Reza Mohammadinejad, Alan Prem Kumar
Summary: CRISPR-Cas technology, originally found in archaea and bacteria, has been repurposed for use in eukaryotic cells, leading to a revolution in genome engineering. Its versatility and feasibility make it the most suitable tool for discovering and studying specific gene mechanisms.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Biotechnology & Applied Microbiology
Xiaowen Huang, Aifang Li, Peng Xu, Yangfan Yu, Shuxuan Li, Lina Hu, Shuying Feng
Summary: Extracellular vesicles (EVs) show promise as a gene delivery platform for targeted CRISPR/Cas systems, overcoming challenges associated with toxicity and immunogenicity. This review explores strategies to improve loading capacity, safety, stability, targeting, and tracking for EV-based CRISPR/Cas delivery. It hypothesizes future avenues for EV-based delivery systems to bridge the gap between gene editing technologies and gene therapies.
JOURNAL OF NANOBIOTECHNOLOGY
(2023)
Review
Biochemistry & Molecular Biology
Yu-Fan Chuang, Andrew J. Phipps, Fan-Li Lin, Valerie Hecht, Alex W. Hewitt, Peng-Yuan Wang, Guei-Sheung Liu
Summary: The CRISPR/Cas system is a groundbreaking genetic technology that allows gene modification by targeting specific genomic sites. Despite its versatility, the translation of CRISPR/Cas system still faces hurdles that need to be overcome, especially in improving delivery efficiency for potential applications in various fields.
CELLULAR AND MOLECULAR LIFE SCIENCES
(2021)
Review
Biotechnology & Applied Microbiology
Kyungmin Kang, Youngjae Song, Inho Kim, Tae-Jung Kim
Summary: The CRISPR-Cas system has revolutionized genetic engineering due to its simplicity, stability, and precision. It has found applications in various fields, including basic research, medical diagnosis and treatment. This review focuses on medical applications, discussing the latest treatment trends and limitations, with a particular emphasis on CRISPR-based therapies for infectious disease, oncology, and genetic disease, as well as diagnostics, screening, immunotherapy, and cell therapy.
BIOENGINEERING-BASEL
(2022)
Article
Microbiology
Anoop Singh, Mohita Gaur, Vishal Sharma, Palak Khanna, Ankur Bothra, Asani Bhaduri, Anupam Kumar Mondal, Debasis Dash, Yogendra Singh, Richa Misra
Summary: This study conducted a comparative analysis of 141 mycobacterial genomes and found the exclusive presence of the CRISPR-Cas III-A system in the Mycobacterium tuberculosis complex. Phylogenetic evidence suggests that this system was acquired through horizontal gene transfer from a Firmicutes bacterium. The variations in CRISPR-Cas organization in M. tuberculosis lineages, particularly due to IS6110 transposition, were also identified.
Review
Infectious Diseases
Shuan Tao, Huimin Chen, Na Li, Wei Liang
Summary: This article discusses the potential importance of the CRISPR-Cas system in controlling antibiotic resistance and its application prospects in preventing and controlling the spread of bacterial drug resistance.
INFECTION AND DRUG RESISTANCE
(2022)
Review
Biotechnology & Applied Microbiology
Tien Van Vu, Duong Thi Hai Doan, Jihae Kim, Yeon Woo Sung, Mil Thi Tran, Young Jong Song, Swati Das, Jae-Yean Kim
Summary: The ultimate goals of genetic engineering are to achieve gene editing and allele introgression with absolute precision and control. Various approaches have been developed in plants for precision genome editing, with significant advancements in HR-mediated editing frequencies through CRISPR/Cas technology. Despite this progress, further research is needed for commercially viable applications of precise genome editing, potentially requiring alternative innovative methods.
PLANT BIOTECHNOLOGY JOURNAL
(2021)
Review
Biochemistry & Molecular Biology
Junhao Wu, Yong Tao, Di Deng, Zhaoli Meng, Yu Zhao
Summary: Hearing loss can be caused by various genetic factors, either in isolation or in association with other symptoms or abnormalities. There is currently no gene therapy available for restoring or improving hearing. The development of the CRISPR/Cas system has provided a promising tool for genetic hearing loss research, and some studies have shown therapeutic efficacy in specific cases. This review discusses the progress in CRISPR/Cas technique, understanding of genetic hearing loss, recent achievements in disease modeling and therapeutic strategies, and the challenges in applying CRISPR/Cas in future clinical treatments.
CELL AND BIOSCIENCE
(2023)
Review
Biotechnology & Applied Microbiology
Wenqian Li, Can Huang, Jingyu Chen
Summary: CRISPR/Cas system is a widely used genome editing tool, but its off-target effects, cytotoxicity, and low efficiency hinder its application in new species. Therefore, system optimization is necessary to improve targeted specificity, reduce toxicity, and enhance editing efficiency.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2022)
Review
Chemistry, Multidisciplinary
Eman A. Taha, Joseph Lee, Akitsu Hotta
Summary: The discovery of CRISPR genome editing technology has provided a versatile approach for treating multiple diseases. However, the safe and effective delivery of genome-editing components remains a key challenge for in vivo genome editing therapy. Recent developments in transient delivery systems and ongoing clinical trials offer potential solutions and applications for in vivo genome editing therapy.
JOURNAL OF CONTROLLED RELEASE
(2022)
Article
Plant Sciences
Michelle Roenspies, Holger Puchta
Summary: A major challenge in plant breeding is the establishment or breakage of genetic linkages through random meiotic recombination. This problem can be addressed by utilizing CRISPR-Cas-mediated chromosome engineering. Researchers found that by inverting a segment of chromosome 2 in Arabidopsis thaliana, they were able to effectively suppress genetic crossovers in that region.
Article
Biotechnology & Applied Microbiology
Sung-Ah Hong, Jung Hwa Seo, Soohyun Wi, Eul Sik Jung, Jihyeon Yu, Gue-Ho Hwang, Ji Hea Yu, Ahreum Baek, Soeon Park, Sangsu Bae, Sung-Rae Cho
Summary: This study successfully increased the expression levels of the ABCD1 gene and reduced the levels of metabolites associated with ADLD using gene editing strategies in cells and mouse models derived from ADLD patients. These findings suggest that gene editing could be a promising therapeutic strategy for treating ADLD.
Article
Biochemistry & Molecular Biology
Omer Habib, Gizem Habib, Gue-Ho Hwang, Sangsu Bae
Summary: Prime editing is a precise and versatile genome editing technique that can directly copy desired genetic modifications into target DNA sites. It has great potential for gene function analysis, disease modeling, and correction of pathogenic mutations. In this study, prime editing was successfully tested in human pluripotent stem cells (hPSCs) and compared with base editing. Whole-genome sequencing showed that prime editing does not lead to guide RNA-independent off-target mutations in the genome.
NUCLEIC ACIDS RESEARCH
(2022)
Article
Engineering, Biomedical
Ju-Chan Park, Jumee Kim, Hyun-Ki Jang, Seung-Yeon Lee, Keun-Tae Kim, Eun-Ji Kwon, Seokwoo Park, Hyun Sik Lee, Hyewon Choi, Seung-Yeol Park, Hee-Jung Choi, Soon-Jung Park, Sung-Hwan Moon, Sangsu Bae, Hyuk-Jin Cha
Summary: This study successfully established multiple isogenic disease models of GNE myopathy by using base editors to construct hPSCs. Different mutations in different domains of the GNE gene were found to cause mutation-specific hyposialylation and gene expression changes, which were closely related to pathological clinical phenotypes. Furthermore, treatment with a drug candidate currently under clinical trials showed a mutation-specific drug response in GNE myopathy disease models.
Article
Medicine, Research & Experimental
Ju-Chan Park, Hyeon-Ki Jang, Jumee Kim, Jun Hee Han, Youngri Jung, Keuntae Kim, Sangsu Bae, Hyuk-Jin Cha
Summary: Precise genome editing of human pluripotent stem cells (hPSCs) is essential for both basic science and biomedical applications. This study demonstrated that high expression of DNA glycosylases, such as uracil DNA glycosylase (UNG), in human embryonic stem cells (hESCs) significantly reduced the editing efficiency of cytosine base editor (CBE) and affected the purity of C to T base substitutions. Transient knockdown of UNG improved the editing efficiency and product purity in hESCs.
MOLECULAR THERAPY-NUCLEIC ACIDS
(2022)
Article
Biotechnology & Applied Microbiology
Sung-Ah Hong, Song-Ee Kim, A-Young Lee, Gue-Ho Hwang, Jong Hoon Kim, Hiroaki Iwata, Soo-Chan Kim, Sangsu Bae, Sang Eun Lee
Summary: In this study, researchers identified and corrected pathogenic mutations in the COL7A1 gene using adenine base editors and prime editors in patients with recessive dystrophic epidermolysis bullosa (RDEB). The edited patient-derived skin equivalents showed positive outcomes in terms of C7 deposition and anchoring fibril formation, suggesting the feasibility of ex vivo gene editing as a potential treatment for RDEB.
News Item
Biochemistry & Molecular Biology
Beomjong Song, Sangsu Bae
Summary: A TnpB-based adenine base editor has been developed and can be loaded into a single AAV vector without compromising editing activity, showing potential for efficient gene editing.
NATURE CHEMICAL BIOLOGY
(2022)
Article
Biochemistry & Molecular Biology
Jong Hee Kim, Jihyeon Yu, Hee Kyoung Kim, Jin Young Kim, Me-Sun Kim, Yong-Gu Cho, Sangsu Bae, Kwon Kyoo Kang, Yu Jin Jung
Summary: Researchers have successfully conducted targeted mutagenesis and golden SNP replacement of the LcyE gene in rice using CRISPR/Cas9 and geminiviral replicon. This breakthrough has the potential to replace all genes within one generation and greatly improve agriculturally important traits.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biochemistry & Molecular Biology
Jin Young Kim, Jong Hee Kim, Young Hee Jang, Jihyeon Yu, Sangsu Bae, Me-Sun Kim, Yong-Gu Cho, Yu Jin Jung, Kwon Kyoo Kang
Summary: In this study, a tomato SGR1-knockout null line was generated using gene editing technology, and it was found that the SGR1 knockout mutants exhibited a turbid brown color in the fruit, with significantly higher levels of chlorophyll and carotenoids compared to the wild-type. Differential gene expression analysis identified 728 differentially expressed genes, most of which were related to photosynthesis, chloroplasts, and carotenoid biosynthesis. These results provide new evidence for understanding the function of SGR1 and the molecular pathways involved in photosynthesis, chloroplasts, and carotenoid biosynthesis.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Plant Sciences
Hee Kyoung Kim, Jin Young Kim, Jong Hee Kim, Ji Yun Go, Yoo-Seob Jung, Hyo Ju Lee, Mi-Jeong Ahn, Jihyeon Yu, Sangsu Bae, Ho Soo Kim, Sang-Soo Kwak, Me-Sun Kim, Yong-Gu Cho, Yu Jin Jung, Kwon Kyoo Kang
Summary: We generated an orange-colored rice callus line by targeted mutagenesis of the orange gene (OsOr) using the CRISPR-Cas9 system. The OC line accumulated higher levels of lutein, beta-carotene, and two beta-carotene isomers compared to the WT line. The expression levels of carotenoid biosynthesis genes were also altered in the OC line.
Correction
Biochemistry & Molecular Biology
Beomjong Song, Sangsu Bae
NATURE CHEMICAL BIOLOGY
(2023)
Article
Chemistry, Analytical
Siheon Seong, Sewon Lee, Sunghyun Bae, Minjae Lee
Summary: This paper presents a fully-differential touch screen panel (TSP) self-capacitance sensing (SCS) system with a self-capacitance mismatch calibration technique. Due to the self-capacitance mismatch of TSP, the SNR loss for the TSP SCS system is caused by the degradation of the AFE dynamic range and gain limitations. The proposed calibration method effectively relieves the mismatch effect in terms of area and power consumption, restoring the SNR by 19.54 dB even under the worst self-capacitance mismatch case.
Review
Chemistry, Analytical
Sunghyun Bae, Hyeon-June Kim
Summary: This paper proposes the use of a two-channel optical time-division multiplexed system with multilevel pulse-amplitude modulation to achieve >100 Gb/s data-center interconnects. Unlike the conventional four-channel system, the two-channel system can be implemented cost-effectively using a wide pulse and integrated transmitter. The paper reviews the current research on the two-channel system and discusses future directions, highlighting the ability to transmit 200 Gb/s signals over standard single-mode fiber using phase-alternating pulses.
Review
Geosciences, Multidisciplinary
Sang-Hun Lee, Sunghyun Bae
Summary: This study focuses on the emission and dispersion of ammonia from livestock manure facilities, as well as on the treatment and recovery of ammonia. It recommends the use of low-cost monitoring sensors integrated with information technology to improve data quality. Emission factors should be classified based on various scenarios and conditions.
GEOSYSTEM ENGINEERING
(2022)