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- CRISPR-Cas technologies: Emerging tools from research to clinical application
- Hana Hyeon, Soonhye Hwang, Yongyang Luo, Eunkyoung Shin, Ji-Hyun Yeom, Hong-Man Kim, Minkyung Ryu, Kangseok Lee
- J. Microbiol. 2025;63(8):e2504012. Published online August 31, 2025
- DOI: https://doi.org/10.71150/jm.2504012
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Abstract
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CRISPR-Cas technologies have emerged as powerful and versatile tools in gene therapy. In addition to the widely used SpCas9 system, alternative platforms including modified amino acid sequences, size-optimized variants, and other Cas enzymes from diverse bacterial species have been developed to apply this technology in various genetic contexts. In addition, base editors and prime editors for precise gene editing, the Cas13 system targeting RNA, and CRISPRa/i systems have enabled diverse and adaptable approaches for genome and RNA editing, as well as for regulating gene expression. Typically, CRISPR-Cas components are transported to the target in the form of DNA, RNA, or ribonucleoprotein complexes using various delivery methods, such as electroporation, adeno-associated viruses, and lipid nanoparticles. To amplify therapeutic efficiency, continued developments in targeted delivery technologies are required, with increased safety and stability of therapeutic biomolecules. CRISPR-based therapeutics hold an inexhaustible potential for the treatment of many diseases, including rare congenital diseases, by making permanent corrections at the genomic DNA level. In this review, we present various CRISPR-based tools, their delivery systems, and clinical progress in the CRISPR-Cas technology, highlighting its innovative prospects for gene therapy.
- [Protocol] Development of DNA aptamers specific for small therapeutic peptides using a modified SELEX method
- Jaemin Lee , Minkyung Ryu , Dayeong Bae , Hong-Man Kim , Seong-il Eyun , Jeehyeon Bae , Kangseok Lee
- J. Microbiol. 2022;60(7):659-667. Published online June 22, 2022
- DOI: https://doi.org/10.1007/s12275-022-2235-4
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- 6 Web of Science
- 6 Crossref
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Abstract
- Aptamers are short single-stranded DNA or RNA oligonucleotides capable of binding with high affinity and specificity to target molecules. Because of their durability and ease of synthesis, aptamers are used in a wide range of biomedical fields, including the diagnosis of diseases and targeted delivery of therapeutic agents. The aptamers were selected using a process called systematic evolution of ligands by exponential enrichment (SELEX), which has been improved for various research purposes since its development in 1990. In this protocol, we describe a modified SELEX method that rapidly produces high aptamer screening yields using two types of magnetic beads. Using this method, we isolated an aptamer that specifically binds to an antimicrobial peptide. We suggest that by conjugating a small therapeutic-specific aptamer to a gold nanoparticle-based delivery system, which enhances the stability and intracellular delivery of peptides, aptamers selected by our method can be used for the development of therapeutic agents utilizing small therapeutic peptides.
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Citations
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- Recent approaches in the application of antimicrobial peptides in food preservation
Satparkash Singh, Bhavna Jha, Pratiksha Tiwari, Vinay G. Joshi, Adarsh Mishra, Yashpal Singh Malik
World Journal of Microbiology and Biotechnology.2024;[Epub] CrossRef - Design and application of microfluidics in aptamer SELEX and Aptasensors
Shikun Zhang, Yingming Zhang, Zhiyuan Ning, Mengxia Duan, Xianfeng Lin, Nuo Duan, Zhouping Wang, Shijia Wu
Biotechnology Advances.2024; 77: 108461. CrossRef - Nanogenosensors based on aptamers and peptides for bioelectrochemical cancer detection: an overview of recent advances in emerging materials and technologies
Babak Mikaeeli Kangarshahi, Seyed Morteza Naghib
Discover Applied Sciences.2024;[Epub] CrossRef - Recent progress of SELEX methods for screening nucleic acid aptamers
Chao Zhu, Ziru Feng, Hongwei Qin, Lu Chen, Mengmeng Yan, Linsen Li, Feng Qu
Talanta.2024; 266: 124998. CrossRef - Aptamer-conjugated gold nanoparticles platform as the intracellular delivery of antibodies for cancer therapy
Ji-Hyun Yeom, Eunkyoung Shin, Hanyong Jin, Haifeng Liu, Yongyang Luo, Youngwoo Nam, Minkyung Ryu, Wooseok Song, Heeyoun Chi, Jeongkyu Kim, Kangseok Lee, Jeehyeon Bae
Journal of Industrial and Engineering Chemistry.2023; 126: 480. CrossRef - Regulation of transforming growth factor-β signaling as a therapeutic approach to treating colorectal cancer
Jana Maslankova, Ivana Vecurkovska, Miroslava Rabajdova, Jana Katuchova, Milos Kicka, Michala Gayova, Vladimir Katuch
World Journal of Gastroenterology.2022; 28(33): 4744. CrossRef
- Recent approaches in the application of antimicrobial peptides in food preservation
- Functional Analysis of the Invariant Residue G791 of Escherichia coli 16S rRNA
- Woo-Seok Song , Hong-Man Kim , Jae-Hong Kim , Se-Hoon Sim , Sang-Mi Ryou , Sanggoo Kim , Chang-Jun Cha , Philip R. Cunningham , Jeehyeon Bae , Kangseok Lee
- J. Microbiol. 2007;45(5):418-421.
- DOI: https://doi.org/2595 [pii]
- 135 View
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Abstract
- The nucleotide at position 791(G791) of E. coli 16S rRNA was previously identified as an invariant residue for ribosomal function. In order to characterize the functional role of G791, base substitutions were introduced at this position, and mutant ribosomes were analyzed with regard to their protein synthesis ability, via the use of a specialized ribosome system. These ribosomal RNA mutations attenuated the ability of ribosomes to conduct protein synthesis by more than 65%. A transition mutation (G to A) exerted a moderate effect on ribosomal function, whereas a transversion mutation (G to C or U) resulted in a loss of protein synthesis ability of more than 90%. The sucrose gradient profiles of ribosomes and primer extension analysis showed that the loss of protein-synthesis ability of mutant ribosomes harboring a base substitution from G to U at position 791 stems partially from its inability to form 70S ribosomes. These findings show the involvement of the nucleotide at position 791 in the association of ribosomal subunits and protein synthesis steps after 70S formation, as well as the possibility of using 16S rRNA mutated at position 791 for the selection of second-site revertants in order to identify ligands that interact with G791 in protein synthesis.
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