Article
Biochemistry & Molecular Biology
Haokun Zhang, Qinlinglan Zhou, Hongyan Chen, Daru Lu
Summary: This study successfully utilized PE3 to induce beta-thalassemia mutations in HUDEP-2 cells, achieving editing ratios up to 100% without off-target effects. Analysis of editing tools will contribute to improved PE3 design in future studies. The characterization of HUDEP-2 beta-thal cells provides ideal models for future therapeutic research.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Multidisciplinary Sciences
Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer, Yan G. Zhao, Karthikeyan Ponnienselvan, Aamir Mir, Erik J. Sontheimer, Guangping Gao, Terence R. Flotte, Scot A. Wolfe, Wen Xue
Summary: The study introduces an NLS-optimized SpCas9-based prime editor that enhances genome editing efficiency and demonstrates the potential to induce tumor formation and correct pathogenic mutations in adult mice through somatic cell editing. The use of dual adeno-associated virus (AAVs) for delivery of a split-intein prime editor further establishes the capability of this system for in vivo installation of sequence modifications with important implications for disease modeling and correction.
NATURE COMMUNICATIONS
(2021)
Article
Biochemistry & Molecular Biology
Haokun Zhang, Ruilin Sun, Jian Fei, Hongyan Chen, Daru Lu
Summary: This study demonstrates the successful correction of a β-thalassemia mutation using prime editing, which restored the normal phenotype and proved the genomic safety of PE3.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Multidisciplinary Sciences
Andrea J. Slavney, Takeshi Kawakami, Meghan K. Jensen, Thomas C. Nelson, Aaron J. Sams, Adam R. Boyko
Summary: The study identified multiple genetic factors influencing coat pheomelanin intensity in domestic dogs, showcasing the complex multigenic nature of the trait. Through genome-wide association studies, new loci related to pheomelanin intensity were discovered, with a linear model explaining over 70% of the variation across different dog breeds.
Editorial Material
Plant Sciences
Niaz Ahmad, Muhammad Jawad Akbar Awan, Shahid Mansoor
Summary: Prime editing allows targeted changes in the genome without double-stranded breaks. However, its efficiency in plants is low. A recent study by Zong et al. improved prime-editing efficiency in both protoplasts and transgenic plants through a simple strategy.
TRENDS IN PLANT SCIENCE
(2023)
Article
Multidisciplinary Sciences
Xiangyang Li, Guiquan Zhang, Shisheng Huang, Yao Liu, Jin Tang, Mingtian Zhong, Xin Wang, Wenjun Sun, Yuan Yao, Quanjiang Ji, Xiaolong Wang, Jianghuai Liu, Shiqiang Zhu, Xingxu Huang
Summary: Strategies to improve the specificity of nuclease-based prime editor (PEn) are needed. Here the authors report a 53BP1-inhibitory ubiquitin variant-assisted PEn platform (uPEn) to inhibit NHEJ and enable precise prime editing for generation of insertions, deletions, and replacements.
NATURE COMMUNICATIONS
(2023)
Article
Medicine, Research & Experimental
Gayoung Jang, Ha Rim Shin, Hyo-Sang Do, Jiyeon Kweon, Soojin Hwang, Soyoung Kim, Sun Hee Heo, Yongsub Kim, Beom Hee Lee
Summary: This study utilized CRISPR-mediated base editors (BEs) and prime editors (PEs) to generate LNS-associated disease models and correct the disease models, suggesting a potential therapeutic strategy for this rare genetic disease.
MOLECULAR THERAPY-NUCLEIC ACIDS
(2023)
Article
Biotechnology & Applied Microbiology
Yichao Li, Jingjing Chen, Shengdar Q. Tsai, Yong Cheng
Summary: Easy-Prime is a machine learning-based program that provides optimized PE design for a wide range of GWAS variants, including factors like RNA folding structure, to enhance editing efficiency.
Article
Biotechnology & Applied Microbiology
Yuan Zong, Yijing Liu, Chenxiao Xue, Boshu Li, Xiangyang Li, Yanpeng Wang, Ji Li, Guanwen Liu, Xingxu Huang, Xiaofeng Cao, Caixia Gao
Summary: In this study, we have improved the editing efficiency of prime editing by engineering the structure of the prime editor and combining it with specific guide RNAs. The optimized prime editor showed significantly increased editing efficiency in plant cells, and when combined with engineered guide RNAs, it resulted in the generation of herbicide-tolerant rice plants.
NATURE BIOTECHNOLOGY
(2022)
Article
Veterinary Sciences
Evzen Korec, Lenka Ungrova, Jiri Hejnar, Adela Grieblova
Summary: This study identified four SNPs associated with longevity in Cane corso dogs, located in genes TDRP, MC2R, FBXO25 and FBXL21. The longevity-associated SNP in FBXL21 was found to localize to the exon, with tryptophan replacing arginine in the protein of long-lived dogs.
BMC VETERINARY RESEARCH
(2022)
Article
Biotechnology & Applied Microbiology
Zackery A. Ely, Nicolas Mathey-Andrews, Santiago Naranjo, Samuel I. Gould, Kim L. Mercer, Gregory A. Newby, Christina M. Cabana, William M. Rideout III, Grissel Cervantes Jaramillo, Jennifer M. Khirallah, Katie Holland, Peyton B. Randolph, William A. Freed-Pastor, Jessie R. Davis, Zachary Kulstad, Peter M. K. Westcott, Lin Lin, Andrew V. Anzalone, Brendan L. Horton, Nimisha B. Pattada, Sean-Luc Shanahan, Zhongfeng Ye, Stefani Spranger, Qiaobing Xu, Francisco J. Sanchez-Rivera, David R. Liu, Tyler Jacks
Summary: Genetically engineered mouse models have limitations in capturing the full range of genetic lesions that drive human cancer. However, a new CRISPR-Cas9 model has been developed to expand this scope through in vivo prime editing. This system allows for precise engineering of various mutations in cell lines and organoids derived from primary tissues, making it possible to study cancer-associated mutations and complex genetic combinations that are challenging with traditional models.
NATURE BIOTECHNOLOGY
(2023)
Article
Multidisciplinary Sciences
Salla Mikkola, Milla Salonen, Jenni Puurunen, Emma Hakanen, Sini Sulkama, Cesar Araujo, Hannes Lohi
Summary: Aggressive behavior in pet dogs is influenced by factors such as age, gender, fearfulness, body size, companionship, and owner's experience, as well as varying between different breeds. Improvements in owner education and breeding practices could help reduce aggressive behavior towards people, while genetic studies may uncover hereditary factors associated with aggression.
SCIENTIFIC REPORTS
(2021)
Article
Biotechnology & Applied Microbiology
Bin Liu, Xiaolong Dong, Haoyang Cheng, Chunwei Zheng, Zexiang Chen, Tomas C. Rodriguez, Shun-Qing Liang, Wen Xue, Erik J. Sontheimer
Summary: This study reports a split prime editor (sPE) that can efficiently perform genome editing and shows potential therapeutic effects in a mouse model. Compared to previous split prime editors, this system reduces the complexity of the constructs and improves efficiency.
NATURE BIOTECHNOLOGY
(2022)
Article
Hematology
Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Ceacile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio
Summary: Beta-thalassemia is a worldwide common genetic disease caused by mutations affecting beta-globin production. Current gene therapy strategies using lentiviral vectors or CRISPR/Cas9 raise safety concerns. This study developed a base editing strategy using the SpRY-ABE8e base editor to correct the most prevalent BT mutation and showed promising results in restoring beta-globin production and correcting inefficient erythropoiesis. This proof-of-concept study paves the way for the development of a safe and effective autologous gene therapy approach for BT.
Article
Biochemistry & Molecular Biology
Xiujuan Lv, Zheng Zheng, Xiao Zhi, Yilin Zhou, Jineng Lv, Yue Zhou, Binrong Wu, Sixiu Liu, Wei Shi, Zongming Song, Jinling Xu, Jia Qu, Dan Xu, Feng Gu
Summary: This study identified a pathogenic mutation in the RPGR ORF15 gene associated with XLRP and successfully corrected it using an enhanced prime editing system. The findings provide important insights for genetic counseling and prenatal diagnosis, as well as pave the way for prime editing-based gene therapy.
Article
Biotechnology & Applied Microbiology
Sanghoon Lee, Min Hee Jung, Kilyoung Song, Jun-Xue Jin, Anukul Taweechaipaisankul, Geon A. Kim, Hyun Ju Oh, Ok Jae Koo, Se Chang Park, Byeong Chun Lee
Summary: The study successfully established klotho monoallelic knockout porcine fetal fibroblasts, but pigs carrying klotho monoallelic knockout fetuses failed to maintain full-term pregnancy due to decreased klotho expression in placenta, leading to pregnancy loss.
Correction
Biotechnology & Applied Microbiology
Sanghoon Lee, Min Hee Jung, Kilyoung Song, Jun-Xue Jin, Anukul Taweechaipaisankul, Geon A. Kim, Hyun Ju Oh, Ok Jae Koo, Se Chang Park, Byeong Chun Lee
Article
Cell Biology
Jeong Pil Han, Jeong Hyeon Lee, Geon Seong Lee, Ok Jae Koo, Su Cheong Yeom
Summary: This study investigated the correlation between nNOS expression and gastrointestinal tract motility using a stress-induced neonatal maternal separation (NMS) mouse model. Results showed a significant increase in nNOS expression in the large intestine, adrenal gland, and hypothalamus tissues in the NMS model, with a positive correlation between whole gastrointestinal transit time and nNOS expression levels. The study suggested that NMS-induced chronic stress triggers nNOS activation in the hypothalamic-pituitary-adrenal (HPA) axis leading to excessive intestinal motility in the lower GI tract.
Article
Biochemistry & Molecular Biology
Hanna Kim, Ji-Yeong Lee, Soo Jeong Park, Eunsang Kwag, Okjae Koo, Joo-Ho Shin
Summary: PARIS plays a crucial role in HCC by promoting cancer progression through suppressing the transcriptional level of PGC-1 alpha. Modulation of PARIS may serve as a promising therapeutic target for HCC based on experimental findings.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
(2021)
Article
Biochemistry & Molecular Biology
Hayoung Song, Ju-Young Ahn, Fanzhuang Yan, Yidong Ran, Okjae Koo, Geung-Joo Lee
Summary: This study determines the specificity and genetic stability of mutations induced by the CRISPR-Cas9 gene-editing system in tobacco alleles. The results provide empirical evidence about the genetic inheritance of CRISPR-targeted alleles in tobacco transformants and identify potential factors contributing to further mutagenesis.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biotechnology & Applied Microbiology
Dong-Ern Kim, Ji-Hye Lee, Kuk-Bin Ji, Kang-Sun Park, Tae-Young Kil, Okjae Koo, Min-Kyu Kim
Summary: Using SCNT combined with CRISPR-Cas9, genome-edited dogs were successfully generated with mutations at the target locus and altered expression of DJ-1.
Article
Biochemistry & Molecular Biology
Ahyoung Park, Hyun Ju Oh, Kukbin Ji, Eunha Miri Choi, Dongern Kim, Eunyoung Kim, Min Kyu Kim
Summary: This study demonstrates the positive effects of equine amniotic fluid mesenchymal stem cell conditioned medium (eAFMSC-CMp7) on oocyte and embryonic development. eAFMSC-CMp7 improves cytoplasmic maturation, enhances blastocyst quality, and has antioxidant effects.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Cell Biology
Islam M. Saadeldin, Bereket Molla Tanga, Seonggyu Bang, Chaerim Seo, Okjae Koo, Sung Ho Yun, Seung Il Kim, Sanghoon Lee, Jongki Cho
Summary: This study developed a model to improve the attachment of porcine embryos in feeder-free culture conditions using endometrium-derived extracellular vesicles (EVs). By analyzing the cargo contents of EVs and intervening with specific compounds, it was found that embryo attachment and outgrowth formation could be enhanced while reducing apoptosis. These findings suggest that EVs have the potential to improve embryo attachment, and undesired effects can be mitigated through specific interventions.
Article
Agriculture, Dairy & Animal Science
Soo-Young Yum, Woojae Choi, Seokjoong Kim, Goo Jang, Okjae Koo
Summary: Gene integration at site-specific loci, such as pAAVS1, was achieved in porcine fibroblasts using TALEN and CRISPR/Cas9. The efficiency of CRISPR/Cas9 was found to be higher than TALEN. Recombinase-mediated cassette exchange (RMCE) was successfully induced to exchange transgenes at the pAAVS1 locus, demonstrating the usefulness of this technology for porcine transgenesis studies.
ANIMAL BIOTECHNOLOGY
(2023)
Article
Reproductive Biology
Chuang Li, Hyun Ju Oh, Haixing Liu, Min Kyu Kim
Summary: Oxidative stress caused by low temperatures during in vitro preservation decreases the quality of boar spermatozoa. Schisandrin B (Sch-B) has been found to counteract oxidative stress in cells. This study aimed to investigate whether treatment with Sch-B could enhance the quality of boar sperm stored at 17 degrees C. Semen samples were divided into four groups and treated with different concentrations of Sch-B. The results showed that treatment with 0.1 mg/L Sch-B improved sperm motility, membrane integrity, and acrosome integrity compared to the control group. It also reduced lipid peroxidation and increased the fertilization ability of the sperm. The findings suggest that Sch-B treatment protects boar sperm from oxidative stress and improves their fertilization ability during liquid preservation.
Correction
Multidisciplinary Sciences
Dong Ern Kim, Ji Hye Lee, Kuk Bin Ji, Eun Ji Lee, Chuang Li, Hyun Ju Oh, Kang Sun Park, Seung Hoon Lee, Okjae Koo, Min Kyu Kim
SCIENTIFIC REPORTS
(2022)
