Editorial Material
Biochemistry & Molecular Biology
Brett M. Sansbury, Eric B. Kmiec
Summary: The expectations for selectively modifying the human genome have never been higher in the last five years. The development of site-specific nucleases is centerstage in effective molecular therapies, with a focus on correcting genetic mutations that cause cancer and inherited diseases. Homology directed repair, a process for reversing mutations and restoring normal protein function, is a key technique that is being researched for human gene editing.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Medicine, General & Internal
Igor Petkovic, Johannes Bischof, Thomas Kocher, Oliver Patrick March, Bernadette Liemberger, Stefan Hainzl, Dirk Strunk, Anna Maria Raninger, Heide-Marie Binder, Julia Reichelt, Christina Guttmann-Gruber, Verena Wally, Josefina Pinon Hofbauer, Johann Wolfgang Bauer, Ulrich Koller
Summary: In this study, researchers used CRISPR/Cas9 gene editing technique to successfully correct a causal pathogenic frameshift mutation in the COL17A1 gene, which leads to junctional epidermolysis bullosa. By inducing both end-joining repair and HDR-mediated pathways, they achieved efficient gene repair and observed improved cellular functions and structures in the corrected cells.
FRONTIERS IN MEDICINE
(2022)
Article
Genetics & Heredity
Dennis Webster, Alla Bondareva, Staci Solin, Taylor Goldsmith, Lin Su, Nathalia de Lima e Martins Lara, Daniel F. Carlson, Ina Dobrinski
Summary: Pigs, sharing physiological and genetic characteristics with humans, have been used as appropriate preclinical models for studying human diseases and developing innovative treatments. Researchers have demonstrated that CRISPR/Cas9-mediated gene editing in porcine spermatogonia is more efficient than previous methods, enabling precise replication of human disease alleles.
FRONTIERS IN GENETICS
(2021)
Article
Medicine, Research & Experimental
Hiromi Miura, Jurai Imafuku, Aki Kurosaki, Masahiro Sato, Yongjie Ma, Guisheng Zhang, Akiko Mizutani, Kenya Kamimura, Channabasavaiah B. Gurumurthy, Dexi Liu, Masato Ohtsuka
Summary: The CRISPR system is a powerful tool for genome editing that has been widely used in medical research. Researchers have developed and validated DEGFP mouse models for evaluating genome editing tools, demonstrating their utility in assessing both NHEJ and HDR processes. The results suggest that these models can serve as valuable tools for in vivo genome editing evaluation.
MOLECULAR THERAPY-NUCLEIC ACIDS
(2021)
Review
Biochemistry & Molecular Biology
Svetlana A. Smirnikhina, Milyausha I. Zaynitdinova, Vasilina A. Sergeeva, Alexander V. Lavrov
Summary: This article investigates the use of methods that influence the cell cycle in genome editing experiments to improve the efficiency of gene editing. Despite the reversible effects on the cell cycle, caution is still needed to prevent cell mutations and malignant transformation.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Review
Biochemistry & Molecular Biology
Christopher E. Denes, Alexander J. Cole, Yagiz Alp Aksoy, Geng Li, Graham Gregory Neely, Daniel Hesselson
Summary: Genome modification holds great potential for disease prevention or treatment, with CRISPR/Cas9 techniques showing promise in altering disease-relevant genes. Competition among DNA repair pathways can lead to undesirable editing outcomes, but small molecule modulators and engineered CRISPR/Cas proteins have been shown to enhance precision editing efficacy.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Biochemistry & Molecular Biology
Kevin Bloh, Natalia Rivera-Torres
Summary: The mechanism of ssODN-directed gene editing has been a topic of discussion within the field of CRISPR gene editing. The ExACT pathway, based on oligo-driven DNA repair, provides a comprehensive understanding of the different ways DNA repair can occur in the presence of a repair oligonucleotide after CRISPR cleavage, by comparing and challenging other similar DNA repair pathways.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Biochemistry & Molecular Biology
Xinyi Li, Bing Sun, Hongrun Qian, Jinrong Ma, Magdalena Paolino, Zhiying Zhang
Summary: Clustered regulatory interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 nuclease (Cas9) is a highly efficient genome editing tool. However, achieving biallelic editing remains a technical challenge. In this study, the authors developed a novel method to enable biallelic editing using CRISPR/Cas9-mediated homology-directed repair (HDR), and enriched the positively targeted cell clones through an integrated selection system. Experimental results on the APP and PSEN1 genes demonstrated high biallelic editing efficiency using this approach.
JOURNAL OF ZHEJIANG UNIVERSITY-SCIENCE B
(2022)
Article
Biochemistry & Molecular Biology
Guoling Li, Xiaohui Yang, Xinxin Luo, Zhenfang Wu, Huaqiang Yang
Summary: The use of small molecules to synchronize the cell cycle in S and G2/M phases enhances the efficiency of CRISPR/Cas9-mediated homology-directed repair (HDR) in animal cells and embryos. The study reveals the common molecular mechanisms that connect cell cycle progression and HDR activity.
CELL AND BIOSCIENCE
(2023)
Review
Pharmacology & Pharmacy
Adrian B. C. Lee, Meng-How Tan, Christina L. L. Chai
Summary: Using small molecules to increase the efficiency of HDR and decrease NHEJ to improve the efficiency of precise knock-in genome editing.
DRUG DISCOVERY TODAY
(2022)
Article
Plant Sciences
Jonas Blomme, Ward Develtere, Ayse Kose, Julia Arraiza Ribera, Christophe Brugmans, Jessica Jaraba-Wallace, Ward Decaestecker, Debbie Rombaut, Alexandra Baekelandt, Alvaro Daniel Fernandez Fernandez, Frank Van Breusegem, Dirk Inze, Thomas Jacobs
Summary: This study presents a simplified heat stress assay that can effectively increase the mutagenesis efficiency of CRISPR in plants and in some cases improve the recovery of mutant progeny.
Article
Chemistry, Multidisciplinary
Yoo Kyung Kang, Juhee Lee, San Hae Im, Joo Hoon Lee, Juhee Jeong, Duk Ki Kim, Seung Yun Yang, Keehoon Jung, Sang-Gyu Kim, Hyun Jung Chung
Summary: A Cas9 conjugate complex system was developed to induce efficient HDR editing with minimal carrier material. The Cas9-LP complexes showed lower cytotoxicity compared to conventional lipofectamine formulations and achieved efficient base correction of the RFP gene in HEK293T cells.
JOURNAL OF INDUSTRIAL AND ENGINEERING CHEMISTRY
(2021)
Article
Plant Sciences
Ye Tang, Zhennan Zhang, Zhiyuan Yang, Jiahe Wu
Summary: CRISPR/Cas9 and Agrobacterium tumefaciensvirulence proteins work together to achieve precise genome editing via homology directed repair.
JOURNAL OF EXPERIMENTAL BOTANY
(2023)
Review
Virology
Micaela Finney, Joseph Romanowski, Zach N. Adelman
Summary: Programmable gene editing systems like CRISPR-Cas have advanced mosquito genome engineering, but low efficiency of sequence integration at DNA double-strand breaks (DSBs) and a lack of understanding about DSB repair in mosquitoes have limited progress. This review highlights relevant steps of DNA DSB repair choice and promising approaches to enhance HDR in the context of mosquito gene editing.
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).
Article
Biochemistry & Molecular Biology
Nova Fong, Tassa Saldi, Ryan M. Sheridan, Michael A. Cortazar, David L. Bentley
Article
Cell Biology
Christopher C. Ebmeier, Benjamin Erickson, Benjamin L. Allen, Mary A. Allen, Hyunmin Kim, Nova Fong, Jeremy R. Jacobsen, Kaiwei Liang, Ali Shilatifard, Robin D. Dowell, William M. Old, David L. Bentley, Dylan J. Taatjes
Article
Cell Biology
Tassa Saldi, Nova Fong, David L. Bentley
GENES & DEVELOPMENT
(2018)
Article
Cell Biology
Benjamin Erickson, Ryan M. Sheridan, Michael Cortazar, David L. Bentley
GENES & DEVELOPMENT
(2018)
Article
Biochemistry & Molecular Biology
Ryan M. Sheridan, Nova Fong, Angelo D'Alessandro, David L. Bentley
Article
Biochemistry & Molecular Biology
Michael A. Cortazar, Ryan M. Sheridan, Benjamin Erickson, Nova Fong, Kira Glover-Cutter, Kristopher Brannan, David L. Bentley
Article
Oncology
Shanshan Pei, Daniel A. Pollyea, Annika Gustafson, Brett M. Stevens, Mohammad Minhajuddin, Rui Fu, Kent A. Riemondy, Austin E. Gillen, Ryan M. Sheridan, Jihye Kim, James C. Costello, Maria L. Amaya, Anagha Inguva, Amanda Winters, Haobin Ye, Anna Krug, Courtney L. Jones, Biniam Adane, Nabilah Khan, Jessica Ponder, Jeffrey Schowinsky, Diana Abbott, Andrew Hammes, Jason R. Myers, John M. Ashton, Travis Nemkov, Angelo D'Alessandro, Jonathan A. Gutman, Haley E. Ramsey, Michael R. Savona, Clayton A. Smith, Craig T. Jordan
Article
Biochemistry & Molecular Biology
Megan E. Summers, Bradley W. Richmond, Swapna Menon, Ryan M. Sheridan, Jonathan A. Kropski, Sarah A. Majka, M. Mark Taketo, Julie A. Bastarache, James D. West, Stijn De Langhe, Patrick Geraghty, Dwight J. Klemm, Hong Wei Chu, Rachel S. Friedman, Yuankai K. Tao, Robert F. Foronjy, Susan M. Majka
Article
Biology
Shannon M. Walsh, Ryan M. Sheridan, Erin D. Lucas, Thu A. Doan, Brian C. Ware, Johnathon Schafer, Rui Fu, Matthew A. Burchill, Jay R. Hesselberth, Beth Ann Jiron Tamburini
Summary: Traditional techniques for detecting foreign antigens in vivo have limitations, prompting the development of a "molecular tracking device" to better study the distribution and retention of antigens in lymph nodes. By utilizing an antigen conjugated to a nuclease-resistant DNA tag and single-cell mRNA sequencing, antigen abundance in lymph nodes can be quantified. This advancement enables new approaches to study the dissemination of antigen-adjuvant conjugates and to identify mechanisms of antigen acquisition and retention at a cellular level in vivo.
Article
Biochemistry & Molecular Biology
Kathryn S. Carpentier, Ryan M. Sheridan, Cormac J. Lucas, Bennett J. Davenport, Frances S. Li, Erin D. Lucas, Mary K. McCarthy, Glennys Reynoso, Nicholas A. May, Beth A. J. Tamburini, Jay R. Hesselberth, Heather D. Hickman, Thomas E. Morrison
Summary: The scavenger receptor MARCO plays critical roles in controlling viremia during arthritogenic alphavirus infections in mice, limiting viral spread to the bloodstream and clearing alphavirus particles from the circulation. This defense mechanism reduces viral tissue burdens and severity of disease, highlighting the importance of MARCO in viremia control.
Article
Cell Biology
Alexander J. Stemm-Wolf, Eileen T. O'Toole, Ryan M. Sheridan, Jacob T. Morgan, Chad G. Pearson
Summary: Transcriptional control of centriole assembly is crucial for cell division, intracellular trafficking, and cilia. RNA splicing factor SON is specifically required for procentriole assembly by regulating the splicing and expression of genes involved in the microtubule cytoskeleton, centrosome, and centriolar satellites. Additionally, SON plays a key role in the proper splicing and expression of CEP131, which is essential for organizing the trafficking and MT network around centrosomes.
MOLECULAR BIOLOGY OF THE CELL
(2021)
Article
Biochemistry & Molecular Biology
Danielle Y. Bilodeau, Ryan M. Sheridan, Balu Balan, Aaron R. Jex, Olivia S. Rissland
Summary: In G. lamblia, despite pared-down cleavage and polyadenylation machinery, 3' end formation still appears to be an important regulatory step for gene expression.
Article
Immunology
Jacob N. Peterson, Susan A. Boackle, Sophina H. Taitano, Allison Sang, Julie Lang, Margot Kelly, Jeremy T. Rahkola, Anjelica M. Miranda, Ryan M. Sheridan, Joshua M. Thurman, V. Koneti Rao, Raul M. Torres, Roberta Pelanda
Summary: The study found that enhanced differentiation of dual κ B cells into germinal center B cells in MRL/lpr mice was due to heightened response to TLR7, TLR9, and type II IFN signaling. SLE patients had elevated levels of dual-BCR B cells expressing V4-34 autoantibodies, which were associated with abnormal distribution of B cell subsets relevant to autoimmunity.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Oncology
Jessica Finlay-Schultz, Austin E. Gillen, Heather M. Brechbuhl, Joshua J. Ivie, Shawna B. Matthews, Britta M. Jacobsen, David L. Bentley, Peter Kabos, Carol A. Sartorius