Review
Biochemistry & Molecular Biology
Tae Hyeong Kim, Seong-Wook Lee
Summary: Viral infections pose a serious threat to human health and require the development of vaccines and antiviral therapeutic agents. Genome editing technology enables precise modification of genomic sequences and has expanded its applicability in developing therapeutics for viral diseases.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Review
Biochemistry & Molecular Biology
Michelle C. E. Mak, Rijan Gurung, Roger S. Y. Foo
Summary: Cardiovascular diseases, especially coronary artery disease (CAD), are the leading cause of death globally. Atherosclerosis is a major factor in CAD, and genome editing technologies can be used to establish models of atherosclerosis and have therapeutic roles in treating atherosclerosis and the clinical implications of CAD.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Biology
Ilnaz Rahimmanesh, Maryam Boshtam, Shirin Kouhpayeh, Hossein Khanahmad, Arezou Dabiri, Shahrzad Ahangarzadeh, Yasaman Esmaeili, Elham Bidram, Golnaz Vaseghi, Shaghayegh Haghjooy Javanmard, Laleh Shariati, Ali Zarrabi, Rajender S. Varma
Summary: Beta-thalassemia is a heterogeneous blood disorder that can be treated using gene editing tools, but there are still challenges to address, such as off-target effects and immunotoxicity.
Review
Genetics & Heredity
Mehmet Cengiz Baloglu, Yasemin Celik Altunoglu, Pinar Baloglu, Ali Burak Yildiz, Nil Turkolmez, Yelda Ozden ciftci
Summary: Legumes are rich in protein and phytochemicals, and their genetic traits have been improved using genome editing technologies. However, governmental regulatory restrictions present a concern.
FRONTIERS IN GENETICS
(2022)
Review
Cell Biology
Fernando Gomez-Garcia, Raquel Martinez-Pulleiro, Noa Carrera, Catarina Allegue, Miguel A. Garcia-Gonzalez
Summary: This study discusses the genetic causes and phenotypic effects of genetic kidney diseases (GKDs) and describes and compares the application of site-specific nuclease systems (such as ZFNs, TALENs, and CRISPR-Cas9). It demonstrates that these systems can be used to edit the genes causing GKDs and generate models that reflect the genetic abnormalities, thus shedding light on many unknown aspects in the field of GKDs.
Review
Biology
Rakesh Kumar Yadav, Manoj Kumar Tripathi, Sushma Tiwari, Niraj Tripathi, Ruchi Asati, Shailja Chauhan, Prakash Narayan Tiwari, Devendra K. Payasi
Summary: Genome editing aims to revolutionize plant breeding and safeguard the global food supply. Mega nucleases were the first tools used for genome editing, followed by zinc finger nucleases (ZFNs) which are more effective due to their ability to create double-stranded breaks. Clustered regularly interspaced short palindromic repeats (CRISPR) are utilized to improve genetics, biomass production, nutrient usage efficiency, and disease resistance. Genome editing technologies can modify plant genomes effectively without introducing foreign DNA, leading to precise breeding methods. Genome-edited crops hold promise for improving the sustainability and climate resilience of food systems and enhancing resistance to abiotic stressors.
Article
Multidisciplinary Sciences
Young Geun Mok, Ji Min Lee, Eugene Chung, Jaesuk Lee, Kayeong Lim, Sung-Ik Cho, Jin-Soo Kim
Summary: This study presents non-toxic, full-length variants of DddAtox, enabling the use of monomeric DdCBEs for efficient editing of mitochondrial DNA. The monomeric DdCBEs showed different mutation patterns compared to conventional dimeric DdCBEs and allowed base editing at sites with only one TALE protein. Additionally, transfecting mRNA encoding mDdCBEs reduced off-target editing in human mitochondrial DNA.
NATURE COMMUNICATIONS
(2022)
Review
Medicine, General & Internal
Bogdan Doroftei, Ovidiu-Dumitru Ilie, Maria Puiu, Alin Ciobica, Ciprian Ilea
Summary: Infertility is a controversial topic with a genetic basis being recognized as a new perspective for understanding the disease. Research indicates that some genes are dispensable for fertility without affecting host homeostasis, while loss of function in certain genes can lead to moderate to severe phenotypic changes.
Review
Genetics & Heredity
Roya Ramezankhani, Neda Minaei, Mahnaz Haddadi, Shukoofeh Torabi, Mahdi Hesaraki, Hamed Mirzaei, Massoud Vosough, Catherine M. Verfaillie
Summary: Gene correction is a promising strategy to treat genetic diseases, with genome editing tools like CRISPR/Cas9 considered novel technologies capable of correcting genetic aberrations. While the increasing number of clinical trials show encouraging outcomes, challenges remain regarding safety and targeted delivery.
Review
Pharmacology & Pharmacy
Amanda Catalina Ramirez-Phillips, Dexi Liu
Summary: Advancements in understanding human genetics have fueled interest in therapeutic genome editing using engineered nucleases, with CRISPR/Cas9 emerging as a cost-effective and efficient system. Clinical applications of CRISPR/Cas9 include treatments for genetic diseases and cancer, highlighting its potential for targeted genetic modifications and viral infection eradication. Future progress in therapeutic genome editing will rely on improved delivery methods and repair efficiency for site-specific gene modification.
Review
Biochemistry & Molecular Biology
Jochen Dobner, Haribaskar Ramachandran, Andrea Rossi
Summary: Genome editing techniques offer the opportunity for precise treatment of human diseases by manipulating the genomes. Recent breakthroughs have been made and clinical trials employing genome editing-based medicine are underway. However, ethical and regulatory guidelines need to be developed and potential risks and future development of this technology need to be further studied.
FRONTIERS IN BIOSCIENCE-LANDMARK
(2022)
Review
Physiology
Amina Saleem, Muhammad Khawar Abbas, Yongming Wang, Feng Lan
Summary: Research has shown that human pluripotent stem cell-derived cardiomyocytes have significantly advanced the study of cardiovascular disease mechanisms. In recent years, advancements in genome editing techniques have greatly improved the efficiency of cell-based mechanism study and therapy for cardiac diseases.
PFLUGERS ARCHIV-EUROPEAN JOURNAL OF PHYSIOLOGY
(2022)
Article
Multidisciplinary Sciences
Hyunji Lee, Seonghyun Lee, Gayoung Baek, Annie Kim, Beum-Chang Kang, Huiyun Seo, Jin-Soo Kim
Summary: This study demonstrates highly efficient mitochondrial DNA editing using DdCBEs in mouse embryos, creating mitochondrial disease models and showing the potential for treating mitochondrial disorders. The authors show the use of split DddA-derived base editors fused to TALEs in mouse embryos with germline transmission.
NATURE COMMUNICATIONS
(2021)
Review
Cell Biology
Lola Koniali, Carsten W. Lederer, Marina Kleanthous
Summary: Advances in HSC genome editing as a treatment for inherited disorders have shown significant progress, with their versatility and accuracy driving potential therapeutic approaches for diseases. The focus of research is on improving the efficiency of HSC modification, treatment tolerability, and the distribution and affordability of corresponding therapies.
Review
Biochemistry & Molecular Biology
Pin Lyu, Baisong Lu
Summary: Designer nucleases, such as ZFN, TALEN, and CRISPR/Cas, have been widely used for various applications, including mechanistic studies, animal model generation, and gene therapy development. Clinical trials utilizing designer nucleases for treating genetic diseases or cancers have shown promising results. However, potential off-target effects and immune responses are challenges that need to be addressed for in vivo applications, particularly in clinical settings. Short-term expression of designer nucleases is necessary to mitigate these risks, and delivery methods enabling transient expression are being explored. Virus-like particles (VLPs) have gained attention as delivery vehicles for short-term expression of designer nucleases. This review summarizes recent developments in utilizing VLPs for safe delivery of gene editing effectors.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Chemistry, Multidisciplinary
Li Dong, Radek Jankele, Matteo Cornaglia, Thomas Lehnert, Pierre Gonczy, Martin A. M. Gijs
Article
Genetics & Heredity
Katerina Brustikova, David Sedlak, Jana Kubikova, Ctibor Skuta, Katerina Solcova, Radek Malik, Petr Bartunek, Petr Svoboda
FRONTIERS IN GENETICS
(2018)
Article
Biochemistry & Molecular Biology
Sravya Ganesh, Filip Horvat, David Drutovic, Michaela Efenberkova, Dominik Pinkas, Anna Jindrova, Josef Pasulka, Rajan Iyyappan, Radek Malik, Andrej Susor, Kristian Vlahovicek, Petr Solc, Petr Svoboda
NUCLEIC ACIDS RESEARCH
(2020)
Article
Genetics & Heredity
Eliska Taborska, Josef Pasulka, Radek Malik, Filip Horvat, Irena Jenickova, Zoe Jelic Matosevic, Petr Svoboda
Article
Biochemistry & Molecular Biology
Shubhangini Kataruka, Martin Modrak, Veronika Kinterova, Radek Malik, Daniela M. Zeitler, Filip Horvat, Jiri Kanka, Gunter Meister, Petr Svoboda
NUCLEIC ACIDS RESEARCH
(2020)
Review
Plant Sciences
Petr Svoboda
FRONTIERS IN PLANT SCIENCE
(2020)
Article
Biology
Radek Jankele, Rob Jelier, Pierre Goenczy
Summary: In the early embryonic development of C. elegans, unequal first cleavage is essential for successful development, as equalizing sizes results in defects in cell lineage, cell cycle progression, cell positioning, division orientation, and cell fate. Additionally, equalized embryos are more susceptible to external compression.
Article
Genetics & Heredity
Radek Malik, Petr Svoboda
Summary: RNA interference (RNAi) is primarily inactive in mammalian cells, but high RNAi activity exists in mouse oocytes due to a truncated Dicer isoform, Dicer(O). CRISPR-based strategies have been evaluated for epigenetic targeting of the endogenous Dicer gene to restore Dicer(O) expression and RNAi activity in mouse embryonic stem cells. However, reactivation of Dicer(O) expression alone is not sufficient to establish a robust canonical RNAi response.
Article
Cell Biology
Zuzana Loubalova, Helena Fulka, Filip Horvat, Josef Pasulka, Radek Malik, Michiko Hirose, Atsuo Ogura, Petr Svoboda
Summary: The study reveals that the piRNA pathway plays crucial roles in both male and female fertility in golden hamsters, in contrast to previous findings in mice which suggested that piRNAs are essential only for male fertility. This suggests that the piRNA pathway may have adaptive mechanisms that allow it to manage emerging genomic threats and acquire new critical roles in the germline.
NATURE CELL BIOLOGY
(2021)
Article
Biochemistry & Molecular Biology
Shubhangini Kataruka, Veronika Kinterova, Filip Horvat, Marcos Iuri Roos Kulmann, Jiri Kanka, Petr Svoboda
Summary: miRNAs are important negative regulators of gene expression in mammalian cells, but their repressive activity is weak during oocyte and maternal-to-zygotic transition. Certain miRNAs, such as porcine ssc-miR-205 and bovine bta-miR-10b, are exceptions and can efficiently suppress gene expression despite the diluting effect of oocyte growth. The study highlights the importance of establishing a favorable miRNA:mRNA stoichiometry for effective gene suppression.
Article
Biochemistry & Molecular Biology
David Zapletal, Eliska Taborska, Josef Pasulka, Radek Malik, Karel Kubicek, Martina Zanova, Christian Much, Marek Sebesta, Valeria Buccheri, Filip Horvat, Irena Jenickova, Michaela Prochazkova, Jan Prochazka, Matyas Pinkas, Jiri Novacek, Diego F. Joseph, Radislav Sedlacek, Carrie Bernecky, Donal O'Carroll, Richard Stefl, Petr Svoboda
Summary: The DExD/H domain of Dicer plays a crucial structural role in mammalian miRNA biogenesis. It locks Dicer in a closed state, facilitating miRNA precursor selection. Absence or mutations of the DExD/H domain unlock Dicer and activate RNAi.
Article
Biology
Jan Petrzilek, Josef Pasulka, Radek Malik, Filip Horvat, Shubhangini Kataruka, Helena Fulka, Petr Svoboda
Summary: This study describes the evolutionary history of the murine gene D6Ertd527e and its orthologs in the rodent Muroidea superfamily. The gene initially emerged as a long non-coding RNA (lncRNA) expressing gene and acquired protein-coding capacity through mutation events. This evolutionary history provides an example of de novo gene formation, evolution of protein-coding capacity, and gene demise.
Review
Biochemistry & Molecular Biology
David Zapletal, Karel Kubicek, Petr Svoboda, Richard Stefl
Summary: RNase III Dicer plays important biological roles in eukaryotes by generating small RNAs for sequence-specific regulations. RNA interference (RNAi) and microRNA (miRNA) pathways are the major Dicer-dependent mechanisms, which produce distinct types of small RNAs. Structural analyses of animal and plant Dicers have revealed the contributions of different domains to substrate recognition and cleavage, highlighting the ancestral role of siRNA generation and the derived features of miRNA biogenesis. The functional divergence is mainly attributed to a RIG-I-like helicase domain, while the dsRNA-binding domain showcases the impressive functional versatility in Dicer-mediated small RNA biogenesis.
Meeting Abstract
Biochemistry & Molecular Biology
P. Svoboda
Article
Biology
Filip Horvat, Helena Fulka, Radek Jankele, Radek Malik, Ma Jun, Katerina Solcova, Radislav Sedlacek, Kristian Vlahovicek, Richard M. Schultz, Petr Svoboda
LIFE SCIENCE ALLIANCE
(2018)