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
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)
Article
Biotechnology & Applied Microbiology
Shengyao Zhi, Yuxi Chen, Guanglan Wu, Jinkun Wen, Jinni Wu, Qianyi Liu, Yang Li, Rui Kang, Sihui Hu, Jiahui Wang, Puping Liang, Junjiu Huang
Summary: Prime editor (PE) is a new genome editing tool that has the potential to correct the majority of known human genetic disease-related mutations. In this study, split-PEs were constructed and delivered using dual adenoassociated viruses (AAVs), successfully mediating gene editing in human cells and adult mouse retina.
Review
Pharmacology & Pharmacy
Wenhan Yu, Zhijian Wu
Summary: Inherited or multifactorial ocular diseases require novel treatment paradigms, with CRISPR genome editing technology showing great promise. However, the delivery of CRISPR/Cas components to target ocular tissues and cells must consider safety and efficacy. Viral and non-viral vectors are used for delivery, but sustained expression of CRISPR/Cas may lead to immune reactions and off-target editing risks.
ADVANCED DRUG DELIVERY REVIEWS
(2021)
Article
Multidisciplinary Sciences
Gregory A. Newby, Jonathan S. Yen, Kaitly J. Woodard, Thiyagaraj Mayuranathan, Cicera R. Lazzarotto, Yichao Li, Heather Sheppard-Tillman, Shaina N. Porter, Yu Yao, Kalin Mayberry, Kelcee A. Everette, Yoonjeong Jang, Christopher J. Podracky, Elizabeth Thaman, Christophe Lechauve, Akshay Sharma, Jordana M. Henderson, Michelle F. Richter, Kevin T. Zhao, Shannon M. Miller, Tina Wang, Luke W. Koblan, Anton P. McCaffrey, John F. Tisdale, Theodosia A. Kalfa, Shondra M. Pruett-Miller, Shengdar Q. Tsai, Mitchell J. Weiss, David R. Liu
Summary: The study demonstrated successful conversion of sickle cell disease allele into a non-pathogenic variant using adenine base editor, with durable therapeutic effects. The edited HSPCs improved physiological parameters and reduced pathological abnormalities in spleens of mice, indicating the potential for long-lasting and effective treatment for SCD.
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
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)
Review
Biotechnology & Applied Microbiology
Jacob H. Goell, Isaac B. Hilton
Summary: Epigenome editing technologies, particularly those based on CRISPR/Cas, have enabled researchers to program precise modifications to endogenous DNA and histones, enhancing our understanding of the relationship between epigenetic marks and gene expression. These advancements have opened up new ways to explore the role of the epigenome in human health and disease, with ongoing improvements expected to further expand the utility of these technologies in clinical research.
TRENDS IN BIOTECHNOLOGY
(2021)
Article
Biochemistry & Molecular Biology
Ananth Pallaseni, Elin Madli Peets, Jonas Koeppel, Juliane Weller, Thomas Vanderstichele, Uyen Linh Ho, Luca Crepaldi, Jolanda van Leeuwen, Felicity Allen, Leopold Parts
Summary: CRISPR/Cas base editors offer precise control over DNA sequences, but their activity is not fully understood. This study reveals sequence biases in base editing activity, with the flanking nucleotides playing a major role. A machine learning model is developed to predict editing activity, allowing more efficient planning of editing campaigns.
NUCLEIC ACIDS RESEARCH
(2022)
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
Biochemistry & Molecular Biology
Kirti Prasad, Anila George, Nithin Sam Ravi, Kumarasamypet M. Mohankumar
Summary: CRISPR/Cas9 system, developed from bacterial adaptive immune system, has revolutionized genome engineering and made gene therapy closer to reality. Its rapid evolution from double strand breaks to advanced gene regulation applications has greatly benefited medical science.
MOLECULAR BIOLOGY REPORTS
(2021)
Article
Multidisciplinary Sciences
Zheng Li, Zhenhui Zhong, Zhongshou Wu, Patrick Pausch, Basem Al-Shayeb, Jasmine Amerasekera, Jennifer A. Doudna, Steven E. Jackson
Summary: Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) systems are important tools for plant genome engineering. In this study, the hypercompact Cas phi nuclease is shown to generate stably inherited gene edits in Arabidopsis. The Cas phi protein variants, vCas phi and nCas phi, exhibit higher editing efficiency and no off-target editing was observed. The characteristics of Cas phi, such as its small size, T-rich minimal protospacer adjacent motif (PAM), and wide range of working temperatures, make it an excellent supplement to existing plant genome editing systems.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2023)
Review
Pharmacology & Pharmacy
Leilei Fu, Zixiang Li, Yueting Ren, Haiyang Yu, Bo Liu, Yuling Qiu
Summary: Triple negative breast cancer (TNBC) is closely associated with abnormal expression of oncogenes and tumor suppressors. The CRISPR/Cas system, a breakthrough gene-editing tool, has potential therapeutic applications in TNBC. This review summarizes the molecular subtypes of TNBC, discusses the potential applications of the CRISPR/Cas system in TNBC treatment, and explores emerging strategies for precise diagnosis of TNBC using this system. These findings demonstrate the effectiveness of the CRISPR/Cas system as a genome editing tool and its promising role in future therapeutic purposes.
BIOCHEMICAL PHARMACOLOGY
(2023)
Article
Microbiology
Benjamin A. Adler, Tomas Hessler, Brady F. Cress, Arushi Lahiri, Vivek K. Mutalik, Rodolphe Barrangou, Jillian Banfield, Jennifer A. Doudna
Summary: In this study, the defensive capabilities of LbuCas13a protein were analyzed, and it was found to have robust antiviral activity against a wide range of phages. By leveraging LbuCas13a targeting, successful genome editing of multiple phages was achieved, demonstrating the versatility and potency of Cas13a as a generalizable tool for editing diverse biological entities.
NATURE MICROBIOLOGY
(2022)
Review
Medicine, Research & Experimental
Sajad Najafi, Shing Cheng Tan, Shahin Aghamiri, Pourya Raee, Zahra Ebrahimi, Zahra Kargar Jahromi, Yazdan Rahmati, Javid Sadri Nahand, Ahmad Piroozmand, Vahid Jajarmi, Hamed Mirzaei
Summary: The article reviews the research progress of CRISPR-Cas technology in treating viral infections, discusses delivery strategies and challenges, and proposes future research directions.
BIOMEDICINE & PHARMACOTHERAPY
(2022)
Article
Biochemistry & Molecular Biology
Edward M. Kennedy, Anand V. R. Kornepati, Hal P. Bogerd, Bryan R. Cullen
Article
Virology
Edward M. Kennedy, David G. Courtney, Kevin Tsai, Bryan R. Cullen
JOURNAL OF VIROLOGY
(2017)
Article
Biochemistry & Molecular Biology
Brigid Chiyoko Poling, Kevin Tsai, Dong Kang, Linda Ren, Edward M. Kennedy, Bryan R. Cullen
Article
Microbiology
Hal P. Bogerd, Edward M. Kennedy, Adam W. Whisnant, Bryan R. Cullen
Article
Cell Biology
Brook E. Heaton, Edward M. Kennedy, Rebekah E. Dumm, Alfred T. Harding, Matthew T. Sacco, David Sachs, Nicholas S. Heaton
Article
Virology
David S. Hsu, Anand V. R. Kornepati, Wayne Glover, Edward M. Kennedy, Bryan R. Cullen
Article
Biochemistry & Molecular Biology
Kevin Tsai, David G. Courtney, Edward M. Kennedy, Bryan R. Cullen
Article
Microbiology
David G. Courtney, Andrea Chalem, Hal P. Bogerd, Brittany A. Law, Edward M. Kennedy, Christopher L. Holley, Bryan R. Cullen
Article
Microbiology
David G. Courtney, Kevin Tsai, Hal P. Bogerd, Edward M. Kennedy, Brittany A. Law, Ann Emery, Ronald Swanstrom, Christopher L. Holley, Bryan R. Cullen
CELL HOST & MICROBE
(2019)
Article
Oncology
Edward M. Kennedy, Terry Farkaly, Peter Grzesik, Jennifer Lee, Agnieszka Denslow, Jacqueline Hewett, Jeffrey Bryant, Prajna Behara, Caitlin Goshert, Daniel Wambua, Ana De Almeida, Judith Jacques, Damian Deavall, James B. Rottman, Joseph C. Glorioso, Mitchell H. Finer, Brian B. Haines, Christophe Queva, Lorena Lerner
MOLECULAR THERAPY-ONCOLYTICS
(2020)
Article
Oncology
Brian B. Haines, Agnieszka Denslow, Peter Grzesik, Jennifer S. Lee, Terry Farkaly, Jacqueline Hewett, Daniel Wambua, Lingxin Kong, Prajna Behera, Judith Jacques, Caitlin Goshert, Michael Ball, Allison Colthart, Mitchel H. Finer, Melissa W. Hayes, Sonia Feau, Edward M. Kennedy, Lorena Lerner, Christophe Queva
Summary: ONCR-177 is an engineered recombinant oncolytic herpes simplex virus with complementary safety mechanisms and transgenes, showing oncolytic effects on human cancer cells while replication is suppressed in normal cells, and also activating immune cells.
CANCER IMMUNOLOGY RESEARCH
(2021)
Article
Cell Biology
Kevin Tsai, Hal P. Bogerd, Edward M. Kennedy, Ann Emery, Ronald Swanstrom, Bryan R. Cullen
Summary: Previous studies have shown that the addition of m(6)A to viral transcripts can enhance replication and pathogenicity of various viruses, including HIV-1. The mechanisms underlying this effect are largely mediated by m(6)A binding proteins, with YTHDC1 and YTHDF2 playing key roles in regulating HIV-1 gene expression. Unexpectedly, YTHDF2 binding to m(6)A sites on HIV-1 transcripts leads to increased stability, contrasting its destabilizing effect on cellular mRNAs containing m(6)A residues.
GENES & DEVELOPMENT
(2021)
Article
Multidisciplinary Sciences
Edward M. Kennedy, Agnieszka Denslow, Jacqueline Hewett, Lingxin Kong, Ana De Almeida, Jeffrey D. Bryant, Jennifer S. Lee, Judy Jacques, Sonia Feau, Melissa Hayes, Elizabeth L. McMichael, Daniel Wambua, Terry Farkaly, Amal A. Rahmeh, Lauren Herschelman, Danielle Douglas, Jacob Spinale, Sanmit Adhikari, Jessica Deterling, Matt Scott, Brian B. Haines, Mitchell H. Finer, Ted T. Ashburn, Christophe Queva, Lorena Lerner
Summary: The synthetic RNA virus platform provides a new approach for repeated intravenous administration of viral immunotherapy, by formulating synthetic RNA viruses within lipid nanoparticles, showing anti-tumor efficacy even in the presence of neutralizing antibodies against the virus.
NATURE COMMUNICATIONS
(2022)
Correction
Microbiology
Edward M. Kennedy, Hal P. Bogerd, Anand V. R. Kornepati, Dong Kang, Delta Ghoshal, Joy B. Marshall, Brigid C. Poling, Kevin Tsai, Nandan S. Gokhale, Stacy M. Horner, Bryan R. Cullen
CELL HOST & MICROBE
(2017)
Article
Microbiology
David G. Courtney, Edward M. Kennedy, Rebekah E. Dumm, Hal P. Bogerd, Kevin Tsai, Nicholas S. Heaton, Bryan R. Cullen
CELL HOST & MICROBE
(2017)
Article
Virology
Naiqing Xu, Xinen Tang, Xin Wang, Miao Cai, Xiaowen Liu, Xiaolong Lu, Shunlin Hu, Min Gu, Jiao Hu, Ruyi Gao, Kaituo Liu, Yu Chen, Xiufan Liu, Xiaoquan Wang
Summary: This study found that the H9N2 subtype avian influenza virus has a high airborne transmissibility, while the H7N9 virus does not. The Hemagglutinin protein of the H9N2 virus was found to play a key role in replication, stability, and airborne transmission.
Article
Virology
Samar S. Ewies, Sabry M. Tamam, Ahmed S. Abdel-Moneim, Sherin R. Rouby
Summary: Contagious ecthyma (CE) is a highly contagious viral disease of sheep and goats worldwide. The study provided a clinical description of CE and screened for genetic variation in the B2L gene. Infected sheep exhibited anorexia and oral lesions, while inoculated chicken embryos showed pock lesions. The B2L gene was successfully amplified and found to be highly conserved.
Article
Virology
Yigal Farnoushi, Dan Heller, Avishai Lublin
Summary: In recent years, new variants of avian reovirus (ARV) have caused a variety of symptoms in chickens worldwide, including viral arthritis/tenosynovitis. This study analyzed emerging ARV variants in Israel and found significant genetic diversity. Most ARV isolates in Israel belonged to genotypic cluster 5 (GC5). The study suggests that Israel has not experienced the emergence of new ARV variants since the introduction of the live vaccine (ISR-7585), but ongoing monitoring is needed due to the continuous emergence of ARV variants.
Article
Virology
Shigeru Tajima, Michiyo Kataoka, Yuki Takamatsu, Hideki Ebihara, Chang-Kweng Lim
Summary: Yokose virus (YOKV), a bat-associated flavivirus, was found to replicate at a slower rate in mosquito cells compared to other mosquito-borne flaviviruses. Specific nucleotide mutations in the virus were identified to enhance its proliferation ability in mosquito cells.
Article
Virology
Alejandra Borjabad, Baojun Dong, Wei Chao, David J. Volsky, Mary Jane Potash
Summary: This study investigated HIV brain disease using a mouse model, and found that poly I:C can reverse associated cognitive impairment and reduce virus burden. The results also revealed transcriptional changes related to neuronal function and innate immune responses.
Article
Virology
Ching-Hung Lin, Feng-Cheng Hsieh, Meilin Wang, Chieh Hsu, Hsuan-Wei Hsu, Chun-Chun Yang, Cheng-Yao Yang, Hung-Yi Wu
Summary: This study demonstrates that the synthesis of coronavirus subgenomic mRNA is not solely determined by the sequence homology between the leader TRS and TRS-B, but also by the disassociation of the coronavirus polymerase from the viral genome. This finding provides a new insight into the transcription mechanism of coronaviruses.
Article
Virology
Nicholas S. Kron, Benjamin W. Neuman, Sathish Kumar, Patricia L. Blackwelder, Dayana Vidal, Delphina Z. Walker-Phelan, Patrick D. I. Gibbs, Lynne A. Fieber, Michael C. Schmale
Summary: Two recent studies documented the genome of a novel virus in marine animals, finding that the virus is widespread in apparently healthy animals but not highly expressed in neurons. The studies also identified viral replication factories and high levels of defective genomes in chronically infected animals.
Article
Virology
Andrew M. Ramey, Laura C. Scott, Christina A. Ahlstrom, Evan J. Buck, Alison R. Williams, Mia Kim Torchetti, David E. Stallknecht, Rebecca L. Poulson
Summary: We successfully detected and characterized highly pathogenic avian influenza viruses in hunter-harvested wild waterfowl samples from western Alaska. Genomic analysis revealed three independent viral introductions into Alaska. Our findings demonstrate the utility and potential limitations of using molecular processing approaches directly on original swab samples for viral research and monitoring.
Article
Virology
Ting Gong, Dongdong Wu, Yongzhi Feng, Xing Liu, Qi Gao, Xiaoyu Zheng, Zebu Song, Heng Wang, Guihong Zhang, Lang Gong
Summary: This study discovered that quercetin can inhibit PEDV replication both in vivo and in vitro, and alleviate the clinical symptoms and intestinal injury caused by the virus. This provides a new direction for the development of PED antiviral drugs.
Article
Virology
Min Zhu, Hao Zeng, Jianqiao He, Yaohui Zhu, Pingping Wang, Jianing Guo, Jinfan Guo, Huabo Zhou, Yifeng Qin, Kang Ouyang, Zuzhang Wei, Weijian Huang, Ying Chen
Summary: The reassortment between avian H9N2 and Eurasian avian-like (EA) H1N1 viruses may have potentially changed from avian-to-mammals adaptation. This study found that the introduction of EA H1N1 internal genes into H9N2 virus restored the replication capability and resulted in extreme virulence in some cases. This raises new concerns for public health due to the possible coexistence of H9N2 and EA H1N1 viruses in dogs.