Article
Veterinary Sciences
Shuyu Guo, Ge Gao, Cuizhen Zhang, Gang Peng
Summary: This study describes an efficient multiplexed genome editing strategy to generate zebrafish mutants and identified a new candidate gene for hearing loss through behavioral tests and histological examination.
VETERINARY SCIENCES
(2022)
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
Pharmacology & Pharmacy
Masataka Nishiga, Lei S. Qi, Joseph C. Wu
Summary: Advancements in genome editing technology, particularly the CRISPR/Cas9 system, have revolutionized biomedical research and hold great promise for therapeutic applications in treating diseases, including those in the cardiovascular field. Therapeutic genome editing strategies are already being tested in clinical trials for hematopoietic diseases and show potential for treating a wide range of conditions.
ADVANCED DRUG DELIVERY REVIEWS
(2021)
Review
Agriculture, Dairy & Animal Science
Julia Popova, Victoria Bets, Elena Kozhevnikova
Summary: Genome editing has practical applications in farm animals, improving production traits, economic value, and disease resistance. It also has potential in biomedical research and drug production, as well as xenograft donors. Recent advancements in site-specific nucleases and embryological delivery methods have revolutionized transgenesis, providing efficient and reliable tools for genome engineering in agriculture.
Review
Biochemistry & Molecular Biology
Yulin Mu, Chengxiao Zhang, Taihua Li, Feng-Jie Jin, Yun-Ju Sung, Hee-Mock Oh, Hyung-Gwan Lee, Long Jin
Summary: Lactobacillus, important in food production and probiotics, can benefit from CRISPR/Cas9-based genome editing to improve strain efficiency and achieve traceless genome modification.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Review
Cell Biology
Gemma Llargues-Sistac, Laia Bonjoch, Sergi Castellvi-Bel
Summary: The use of next-generation sequencing (NGS) technologies has greatly advanced our understanding of the mutational landscape of complex human diseases such as cancer. Haploid human cell models, such as the HAP1 cell line, have emerged as valuable tools for functional gene studies, especially in combination with CRISPR-Cas9 gene editing technology. This review explores the recent applications of the HAP1 cell line in functional genetic studies and high-throughput genetic screens, highlighting its potential to enhance our understanding of gene function and the genetic basis of human diseases identified through NGS technologies, and its implications for clinical practice and patient care.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2023)
Article
Microbiology
Xueli Zhang, Chaohui Zhang, Caijiao Liang, Bizhou Li, Fanmei Meng, Yuncan Ai
Summary: Bacteriophages, the most abundant organisms in the biosphere, have been sequenced extensively. However, the study of bacteriophage functional genomics has been hindered by a lack of effective research methods. This study designed a phage genome editing platform based on the CRISPR-Cas9 system, and successfully achieved gene editing in Vibrio natriegens phage TT4P2. This platform has the potential to advance research on phage gene diversity and accelerate the development of phage synthetic biology and nanotechnology.
MICROBIOLOGY SPECTRUM
(2022)
Review
Pharmacology & Pharmacy
Siwei Chen, Deng Chen, Bin Liu, Hidde J. Haisma
Summary: CRISPR/Cas9-mediated genome engineering has extensive applications in basic biology, biotechnology, and medicine. However, the low gene modification efficiency and uncontrollable prolonged Cas9 activity hinder its further use. Researchers have explored small molecules with clinical potential to precisely modulate CRISPR/Cas9 genome editing activity.
DRUG DISCOVERY TODAY
(2022)
Article
Chemistry, Multidisciplinary
Weiqi Cai, Ji Liu, Xianghan Chen, Lanqun Mao, Ming Wang
Summary: Researchers have developed a conditional and cell-selective genome editing system called enzyme-inducible CRISPR (eiCRISPR), which is controlled by disease-associated enzymes. This system consists of Cas9 protein, a self-blocked inactive single-guide RNA (bsgRNA), and a chemically caged deoxyribozyme (DNAzyme). By chemically modifying the DNAzyme, the researchers were able to suppress its cleavage ability and selectively activate eiCRISPR for genome editing in cancer cells using tumor cell-overexpressed enzymes. They successfully demonstrated the potential of this system for cancer therapy by editing the human papillomavirus 18 E6 gene in a tumor-bearing xenograft using biodegradable lipid nanoparticles.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
(2022)
Review
Cardiac & Cardiovascular Systems
Yashendra Sethi, Arun Umesh Mahtani, Nimrat Khehra, Inderbir Padda, Neil Patel, Sneha Annie Sebastian, Gurnaaz Malhi, Oroshay Kaiwan, Sunil Saith, Gurpreet Johal
Summary: Cardiac Amyloidosis (CA) is a systemic disorder caused by the deposition of transthyretin (TTR) in the myocardium, leading to various manifestations in the heart. Recent advances have shown a higher prevalence of CA than previously thought. Treatments for TTR cardiac amyloidosis include TTR stabilizers and RNA interference. CRISPR-Cas9 gene editing has shown promise in reducing amyloid accumulation in tissues. Early human trials have demonstrated a significant reduction in serum TTR proteins with CRISPR-Cas9 therapy, suggesting its potential as a curative treatment for CA.
CURRENT PROBLEMS IN CARDIOLOGY
(2023)
Review
Biochemistry & Molecular Biology
Min Ho Lee, Jae Il Shin, Jae Won Yang, Keum Hwa Lee, Do Hyeon Cha, Jun Beom Hong, Yeoeun Park, Eugene Choi, Kalthoum Tizaoui, Ai Koyanagi, Louis Jacob, Seoyeon Park, Ji Hong Kim, Lee Smith
Summary: This review highlights the current and potential use of CRISPR-Cas9 in the management of autoimmune diseases. Several studies have shown that CRISPR-Cas9 can be utilized for immunomodulation, reducing cholesterol, and treating rare diseases. However, there is limited research on the treatment of autoimmune diseases using CRISPR-Cas9.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biotechnology & Applied Microbiology
Kristina J. Tatiossian, Robert D. E. Clark, Chun Huang, Matthew E. Thornton, Brendan H. Grubbs, Paula M. Cannon
Summary: This study found that CRISPR-Cas9 indel signatures can be used to identify gRNAs that maximize HDR outcomes, with the frequency of deletions resulting from MMEJ repair of at least 3 base pairs better predicts HDR frequency. Existing tools can be repurposed to identify gRNAs that promote HDR.
Article
Plant Sciences
Syed Inzimam Ul Haq, Dianfeng Zheng, Naijie Feng, Xingyu Jiang, Feng Qiao, Jin-Sheng He, Quan-Sheng Qiu
Summary: This review summarizes the advances of CRISPR/Cas9 technology in plant genome editing and discusses its applications in forage breeding, as well as the potential and challenges.
JOURNAL OF PLANT PHYSIOLOGY
(2022)
Article
Chemistry, Physical
Juhee Lee, Yoo Kyung Kang, Eonju Oh, Juhee Jeong, San Hae Im, Duk Ki Kim, Haeshin Lee, Sang-Gyu Kim, Keehoon Jung, Hyun Jung Chung
Summary: The study presents a cancer gene therapy strategy based on NanoRNP that efficiently blocks the PD-L1 immune checkpoint and induces an antitumor effect in vivo without the need for combination therapy. In vivo results demonstrate that NanoRNP can induce indels in target cells at high frequencies, significantly suppressing tumor growth.
CHEMISTRY OF MATERIALS
(2022)
Article
Multidisciplinary Sciences
Beate Rieblinger, Hicham Sid, Denise Duda, Tarik Bozoglu, Romina Klinger, Antonina Schlickenrieder, Kamila Lengyel, Krzysztof Flisikowski, Tatiana Flisikowska, Nina Simm, Alessandro Grodziecki, Carolin Perleberg, Andrea Bahr, Lucie Carrier, Mayuko Kurome, Valeri Zakhartchenko, Barbara Kessler, Eckhard Wolf, Lutz Kettler, Harald Luksch, Ibrahim T. Hagag, Daniel Wise, Jim Kaufman, Benedikt B. Kaufer, Christian Kupatt, Angelika Schnieke, Benjamin Schusser
Summary: Research on genetically modified animals has mostly focused on mice, but also includes species like pigs that are more physiologically similar to humans, and cross-species comparisons with phylogenetically distant species like chickens. CRISPR-Cas9 is a versatile genetic editing method applicable across various species. The successful generation of transgenic chickens and pigs that express Cas9 in all organs has confirmed the functionality of Cas9 across different target genes, cell types, and in vivo applications.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2021)
Review
Oncology
Kenneth Aldape, Kevin M. Brindle, Louis Chesler, Rajesh Chopra, Amar Gajjar, Mark R. Gilbert, Nicholas Gottardo, David H. Gutmann, Darren Hargrave, Eric C. Holland, David T. W. Jones, Johanna A. Joyce, Pamela Kearns, Mark W. Kieran, Ingo K. Mellinghoff, Melinda Merchant, Stefan M. Pfister, Steven M. Pollard, Vijay Ramaswamy, Jeremy N. Rich, Giles W. Robinson, David H. Rowitch, John H. Sampson, Michael D. Taylor, Paul Workman, Richard J. Gilbertson
NATURE REVIEWS CLINICAL ONCOLOGY
(2019)
Article
Cell & Tissue Engineering
Miller Huang, Jignesh Tailor, Qiqi Zhen, Aaron H. Gillmor, Matthew L. Miller, Holger Weishaupt, Justin Chen, Tina Zheng, Emily K. Nash, Lauren K. McHenry, Zhenyi An, Fubaiyang Ye, Yasuhiro Takashima, James Clarke, Harold Ayetey, Florence Mg Cavalli, Betty Luu, Branden S. Moriarity, Shirin Ilkhanizadeh, Lukas Chavez, Chunying Yu, Kathreena M. Kurian, Thierry Magnaldo, Nicolas Sevenet, Philipp Koch, Steven M. Pollard, Peter Dirks, Michael P. Snyder, David A. Largaespada, Yoon Jae Cho, Joanna J. Phillips, Fredrik J. Swartling, A. Sorana Morrissy, Marcel Kool, Stefan M. Pfister, Michael D. Taylor, Austin Smith, William A. Weiss
Review
Cell Biology
Faye L. Robertson, Maria-Angeles Marques-Torrejon, Gillian M. Morrison, Steven M. Pollard
DISEASE MODELS & MECHANISMS
(2019)
Review
Oncology
Charles A. C. Williams, Abdenour Soufi, Steven M. Pollard
SEMINARS IN CANCER BIOLOGY
(2020)
Article
Cell & Tissue Engineering
Mantas Matjusaitis, Laura J. Wagstaff, Andrea Martella, Bart Baranowski, Carla Blin, Sabine Gogolok, Anna Williams, Steven M. Pollard
Article
Materials Science, Multidisciplinary
Pedro M. Costa, Kuo-Ching Mei, Martin Kreuzer, Yueting Li, Hosny A. Neveen, Vivien Grant, Frederic Festy, Steven M. Pollard, Khuloud T. Al-Jamal
Article
Cell & Tissue Engineering
Raul Bardini Bressan, Benjamin Southgate, Kirsty M. Ferguson, Carla Blin, Vivien Grant, Neza Alfazema, Jimi C. Wills, Maria Angeles Marques-Torrejon, Gillian M. Morrison, James Ashmore, Faye Robertson, Charles A. C. Williams, Leanne Bradley, Alex von Kriegsheim, Richard A. Anderson, Simon R. Tomlinson, Steven M. Pollard
Summary: Point mutations within histone H3.3 are frequent in pediatric high-grade gliomas (pHGGs), with distinct mutations arising in different brain regions such as the forebrain and hindbrain. Regional differences in the response of fetal neural stem cells to these mutations suggest a possible role in tumorigenesis and tumor development.
Article
Biochemistry & Molecular Biology
Ester Gangoso, Benjamin Southgate, Leanne Bradley, Stefanie Rus, Felipe Galvez-Cancino, Niamh McGivern, Esra Guc, Chantriolnt-Andreas Kapourani, Adam Byron, Kirsty M. Ferguson, Neza Alfazema, Gillian Morrison, Vivien Grant, Carla Blin, IengFong Sou, Maria Angeles Marques-Torrejon, Lucia Conde, Simona Parrinello, Javier Herrero, Stephan Beck, Sebastian Brandner, Paul M. Brennan, Paul Bertone, Jeffrey W. Pollard, Sergio A. Quezada, Duncan Sproul, Margaret C. Frame, Alan Serrels, Steven M. Pollard
Summary: In this study, it was found that GBM stem cells acquire the ability to evade immune clearance through epigenetic immunoediting, leading to the formation of an immunosuppressive tumor microenvironment. This process is not driven by genetic selection but by altering the transcriptional and epigenetic profile of GSCs after immune attack.
Article
Multidisciplinary Sciences
Maria Angeles Marques-Torrejon, Charles A. C. Williams, Benjamin Southgate, Neza Alfazema, Melanie P. Clements, Claudia Garcia-Diaz, Carla Blin, Nerea Arranz-Emparan, Jane Fraser, Noor Gammoh, Simona Parrinello, Steven M. Pollard
Summary: Neural stem cells can transition between states of proliferation and quiescence, with LRIG1 identified as a crucial regulator in maintaining cells in a quiescent state and priming them for cell cycle re-entry and EGFR responsiveness by modulating the EGFR pathway.
NATURE COMMUNICATIONS
(2021)
Article
Multidisciplinary Sciences
Sara Badodi, Nicola Pomella, Xinyu Zhang, Gabriel Rosser, John Whittingham, Maria Victoria Niklison-Chirou, Yau Mun Lim, Sebastian Brandner, Gillian Morrison, Steven M. Pollard, Christopher D. Bennett, Steven C. Clifford, Andrew Peet, M. Albert Basson, Silvia Marino
Summary: The deregulation of chromatin modifiers is found to be essential in the pathogenesis of medulloblastoma. In certain cases, there is a BMI1-dependent sensitivity to deregulation of inositol metabolism. Additionally, the study demonstrates that the combination of IP6 with cisplatin enhances cytotoxicity in medulloblastoma.
NATURE COMMUNICATIONS
(2021)
Article
Multidisciplinary Sciences
Lucy J. Brooks, Melanie P. Clements, Jemima J. Burden, Daniela Kocher, Luca Richards, Sara Castro Devesa, Leila Zakka, Megan Woodberry, Michael Ellis, Zane Jaunmuktane, Sebastian Brandner, Gillian Morrison, Steven M. Pollard, Peter B. Dirks, Samuel Marguerat, Simona Parrinello
Summary: The study reveals that glioblastoma cells infiltrating into the white matter acquire a pre-oligodendrocyte-like fate, leading to decreased proliferation and invasion of tumors. This differentiation is a response to white matter injury, driven by the upregulation of SOX10. Additionally, the study suggests that exploiting this latent program may offer treatment opportunities for a subset of patients.
NATURE COMMUNICATIONS
(2021)
Article
Cell Biology
Mario A. F. Soares, Diogo S. Soares, Vera Teixeira, Abeer Heskol, Raul Bardini Bressan, Steven M. Pollard, Raquel A. Oliveira, Diogo S. Castro
Summary: The differential binding of TFs Brn2 and Ascl1 to mitotic chromosomes, governed by their electrostatic properties, plays an important role in defining the temporal order of transcriptional reactivation during mitosis-to-G1 transition. Competing with Brn2 binding during mitotic exit reduces the transcription of its target gene Nestin. Transcription onset for Brn2 and Ascl1 targets occurs at different phases during mitotic exit.
GENES & DEVELOPMENT
(2021)
Article
Biology
Karin Purshouse, Elias T. Friman, Shelagh Bolyle, Pooran Singh Dewari, Vivien Grant, Alhafidz Hamdan, Gillian M. Morrison, Paul M. Brennan, Sjoerd Beentjes, Steven M. Pollard, Wendy A. Bickmore
Summary: This study used super-resolution imaging and quantitative image analysis and found that ecDNA in GBM stem cells do not routinely cluster or closely interact with transcriptional hubs. The increased copy number of oncogene-harbouring ecDNA is the primary driver of high levels of oncogene transcription, rather than specific interactions with each other or with high concentrations of the transcriptional machinery.
Article
Cell Biology
Katerina Lawlor, Maria Angeles Marques-Torrejon, Gopuraja Dharmalingham, Yasmine El-Azhar, Michael D. Schneider, Steven M. Pollard, Tristan A. Rodriguez
GENES & DEVELOPMENT
(2020)
Article
Cell Biology
Maria Angeles Marques-Torrejon, Ester Gangoso, Steven M. Pollard
DISEASE MODELS & MECHANISMS
(2018)