scholarly journals Use of Customizable Nucleases for Gene Editing and Other Novel Applications

Genes ◽  
2020 ◽  
Vol 11 (9) ◽  
pp. 976
Author(s):  
Pradeep Reddy ◽  
Felipe Vilella ◽  
Juan Carlos Izpisua Belmonte ◽  
Carlos Simón
Keyword(s):  
Animal Models ◽  
Genome Editing ◽  
Biomedical Research ◽  
Laboratory Animal ◽  
Gene Editing ◽  
Genetic Disorders ◽  
Basic Research ◽  
The Past ◽  
Novel Applications ◽  
In Cells

The development of novel genome editing tools has unlocked new opportunities that were not previously possible in basic and biomedical research. During the last two decades, several new genome editing methods have been developed that can be customized to modify specific regions of the genome. However, in the past couple of years, many newer and more exciting genome editing techniques have been developed that are more efficient, precise, and easier to use. These genome editing tools have helped to improve our understanding of genetic disorders by modeling them in cells and animal models, in addition to correcting the disease-causing mutations. Among the genome editing tools, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has proven to be the most popular one due to its versatility and has been successfully used in a wide variety of laboratory animal models and plants. In this review, we summarize the customizable nucleases currently used for genome editing and their uses beyond the modification of genome. We also discuss the potential future applications of gene editing tools for both basic research and clinical purposes.

scholarly journals Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

2020 ◽  
Vol 5 (1) ◽  
Author(s):  
Hongyi Li ◽  
Yang Yang ◽  
Weiqi Hong ◽  
Mengyuan Huang ◽  
Min Wu ◽  
...  
Keyword(s):  
Animal Models ◽  
Genome Editing ◽  
Gene Editing ◽  
Basic Research ◽  
Human Diseases ◽  
Eukaryotic Cells ◽  
Zinc Finger Nucleases ◽  
Transcription Activator ◽  
Major Genome ◽  
Almost All

AbstractBased on engineered or bacterial nucleases, the development of genome editing technologies has opened up the possibility of directly targeting and modifying genomic sequences in almost all eukaryotic cells. Genome editing has extended our ability to elucidate the contribution of genetics to disease by promoting the creation of more accurate cellular and animal models of pathological processes and has begun to show extraordinary potential in a variety of fields, ranging from basic research to applied biotechnology and biomedical research. Recent progress in developing programmable nucleases, such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR)–Cas-associated nucleases, has greatly expedited the progress of gene editing from concept to clinical practice. Here, we review recent advances of the three major genome editing technologies (ZFNs, TALENs, and CRISPR/Cas9) and discuss the applications of their derivative reagents as gene editing tools in various human diseases and potential future therapies, focusing on eukaryotic cells and animal models. Finally, we provide an overview of the clinical trials applying genome editing platforms for disease treatment and some of the challenges in the implementation of this technology.

scholarly journals Genome editing in large animals: current status and future prospects

2019 ◽  
Vol 6 (3) ◽  
pp. 402-420 ◽  
Author(s):  
Jianguo Zhao ◽  
Liangxue Lai ◽  
Weizhi Ji ◽  
Qi Zhou
Keyword(s):  
Regenerative Medicine ◽  
Genome Editing ◽  
Biomedical Research ◽  
Human Disease ◽  
Gene Editing ◽  
Disease Modeling ◽  
Current Status ◽  
Future Prospects ◽  
Recent Advances ◽  
Large Animals

AbstractLarge animals (non-human primates, livestock and dogs) are playing important roles in biomedical research, and large livestock animals serve as important sources of meat and milk. The recently developed programmable DNA nucleases have revolutionized the generation of gene-modified large animals that are used for biological and biomedical research. In this review, we briefly introduce the recent advances in nuclease-meditated gene editing tools, and we outline these editing tools’ applications in human disease modeling, regenerative medicine and agriculture. Additionally, we provide perspectives regarding the challenges and prospects of the new genome editing technology.

scholarly journals Embryo-Based Large Fragment Knock-in in Mammals: Why, How and What’s Next

Genes ◽  
2020 ◽  
Vol 11 (2) ◽  
pp. 140 ◽  
Author(s):  
Steven Erwood ◽  
Bin Gu
Keyword(s):  
Animal Model ◽  
Animal Models ◽  
Genome Editing ◽  
Biomedical Research ◽  
Future Development ◽  
Genetic Modification ◽  
Technological Innovations ◽  
Model Generation ◽  
Large Fragment ◽  
Diverse Range

Endonuclease-mediated genome editing technologies, most notably CRISPR/Cas9, have revolutionized animal genetics by allowing for precise genome editing directly through embryo manipulations. As endonuclease-mediated model generation became commonplace, large fragment knock-in remained one of the most challenging types of genetic modification. Due to their unique value in biological and biomedical research, however, a diverse range of technological innovations have been developed to achieve efficient large fragment knock-in in mammalian animal model generation, with a particular focus on mice. Here, we first discuss some examples that illustrate the importance of large fragment knock-in animal models and then detail a subset of the recent technological advancements that have allowed for efficient large fragment knock-in. Finally, we envision the future development of even larger fragment knock-ins performed in even larger animal models, the next step in expanding the potential of large fragment knock-in in animal models.

Of mice and models: improved animal models for biomedical research

2002 ◽  
Vol 11 (3) ◽  
pp. 115-132 ◽  
Author(s):  
Ernesto Bockamp ◽  
Marko Maringer ◽  
Christian Spangenberg ◽  
Stephan Fees ◽  
Stuart Fraser ◽  
...  
Keyword(s):  
Animal Models ◽  
Mouse Models ◽  
Biomedical Research ◽  
Applied Research ◽  
Mouse Genome ◽  
Gene Knockout ◽  
Genetic Disorders ◽  
Murine Models ◽  
Good State ◽  
Basic And Applied Research

The ability to engineer the mouse genome has profoundly transformed biomedical research. During the last decade, conventional transgenic and gene knockout technologies have become invaluable experimental tools for modeling genetic disorders, assigning functions to genes, evaluating drugs and toxins, and by and large helping to answer fundamental questions in basic and applied research. In addition, the growing demand for more sophisticated murine models has also become increasingly evident. Good state-of-principle knowledge about the enormous potential of second-generation conditional mouse technology will be beneficial for any researcher interested in using these experimental tools. In this review we will focus on practice, pivotal principles, and progress in the rapidly expanding area of conditional mouse technology. The review will also present an internet compilation of available tetracycline-inducible mouse models as tools for biomedical research ( http://www.zmg.uni-mainz.de/tetmouse/ ).

scholarly journals Genome Editing Animals and the Promise of Control in a (Post-) Anthropocentric World

2019 ◽  
Vol 26 (1) ◽  
pp. 3-25
Author(s):  
Rosine Kelz
Keyword(s):  
Genome Editing ◽  
Ecosystem Management ◽  
Biomedical Research ◽  
Gene Editing ◽  
Germ Line ◽  
Life Sciences ◽  
Modern Life ◽  
Daily Lives ◽  
The Public ◽  
Microbiological Research

Gene editing tools are ‘revolutionizing’ microbiological research. Much of the public debate focuses on the possibility of human germ line applications. The use of genome editing to alter non-human animals, however, will have more immediate impacts on our daily lives. Genome edited animals are used for basic biological and biomedical research and could soon play a role in the livestock industry and ecosystem management. Genome editing thus provides an occasion to rethink societal narratives about the relationships between humans and other animals. Even though the technique can be easily incorporated as an example into a conventional storyline about the development of the modern life sciences as striving for control over nature, it can also help to highlight the anthropocentric biases expressed in these narratives and demonstrate the continuities between humans and other animals.

scholarly journals From Gene Targeting to Genome Editing: Transgenic animals applications and beyond

2015 ◽  
Vol 87 (2 suppl) ◽  
pp. 1323-1348 ◽  
Author(s):  
MAURÍCIO ROCHA-MARTINS ◽  
GABRIEL R. CAVALHEIRO ◽  
GABRIEL E. MATOS-RODRIGUES ◽  
RODRIGO A.P. MARTINS
Keyword(s):  
Genome Editing ◽  
Biomedical Research ◽  
Transgenic Animals ◽  
Paradigm Shifts ◽  
New Techniques ◽  
Future Directions ◽  
Cellular Physiology ◽  
The Past ◽  
Genome Modification ◽  
Help Model

ABSTRACTGenome modification technologies are powerful tools for molecular biology and related areas. Advances in animal transgenesis and genome editing technologies during the past three decades allowed systematic interrogation of gene function that can help model how the genome influences cellular physiology. Genetic engineering via homologous recombination (HR) has been the standard method to modify genomic sequences. Nevertheless, nuclease-guided genome editing methods that were developed recently, such as ZFN, TALEN and CRISPR/Cas, opened new perspectives for biomedical research. Here, we present a brief historical perspective of genome modification methods, focusing on transgenic mice models. Moreover, we describe how new techniques were discovered and improved, present the paradigm shifts and discuss their limitations and applications for biomedical research as well as possible future directions.

scholarly journals Gene Editing in Rabbits: Unique Opportunities for Translational Biomedical Research

2021 ◽  
Vol 12 ◽  
Author(s):  
Jie Xu ◽  
Jifeng Zhang ◽  
Dongshan Yang ◽  
Jun Song ◽  
Brooke Pallas ◽  
...  
Keyword(s):  
Animal Model ◽  
Biomedical Research ◽  
Gene Editing ◽  
Gene Knockout ◽  
Ocular Diseases ◽  
The Past ◽  
Research Areas ◽  
Large Size ◽  
Transgenic Rabbits ◽  
Better Than

The rabbit is a classic animal model for biomedical research, but the production of gene targeted transgenic rabbits had been extremely challenging until the recent advent of gene editing tools. More than fifty gene knockout or knock-in rabbit models have been reported in the past decade. Gene edited (GE) rabbit models, compared to their counterpart mouse models, may offer unique opportunities in translational biomedical research attributed primarily to their relatively large size and long lifespan. More importantly, GE rabbit models have been found to mimic several disease pathologies better than their mouse counterparts particularly in fields focused on genetically inherited diseases, cardiovascular diseases, ocular diseases, and others. In this review we present selected examples of research areas where GE rabbit models are expected to make immediate contributions to the understanding of the pathophysiology of human disease, and support the development of novel therapeutics.

scholarly journals Gene Editing: A Double-Edged Sword

2021 ◽  
pp. 727-735
Author(s):  
Akshara K. Raut ◽  
Tripti Waghmare
Keyword(s):  
Animal Models ◽  
Biomedical Research ◽  
Medical Practice ◽  
Gene Editing ◽  
Eukaryotic Cells ◽  
Zinc Finger Nucleases ◽  
Foreseeable Future ◽  
Extreme Hazards ◽  
Programmable Nucleases

This essay is about intrinsic planning parts that can alternate the enlarge of the particle that regulates our herbal cycles, the genome. Since the 1990s, first-class enchantment has been a focal factor of research. It commenced with the genome undertaking and will proceed to be an ambassador for the foreseeable future. The functions are many, and they are anticipated to have a significant speculative effect as properly as extraordinarily extreme hazards. The genome altering development trends have opened up the technique to truly zero in on and exchange genomic progressions in nearly all eukaryotic cells, whether or not they are planned or bacterial nucleases. Genome editing has loosened up our capacity to grant an explanation for the role of inherited qualities in infection with the aid of accelerating the development of increased right smartphone and models of animal of psychotic cycles, and it has begun to exhibit extraordinarily top achievable in a variety of fields, ranging from indispensable look up to utilized biotechnology and biomedical research. The late boom in the development of programmable nucleases, such as zinc-finger nucleases (ZFNs), file activator-like effector nucleases (TALENs), and assembled reliably interspaced quick palindromic repeat (CRISPR)– Cas-related nucleases, has accelerated the transition of fee from idea to medical practice. We observe the purposes of their subordinate reagents as quality-changing units in a range of human illnesses, and anticipated future medicines, which focuses on eukaryotic cells and animal models, in this evaluation of modern-day advances in the three critical genome-modifying propels (ZFNs, TALENs, and CRISPR/Cas9). Finally, we have a framework for clinical primers to use genome adjusting phases for sickness therapy, as nicely as some of the difficulties encountered throughout implementation.

scholarly journals Synthetic Biology Brings New Challenges to Managing Biosecurity and Biosafety

2021 ◽  
pp. 117-129
Author(s):  
Andrew Jin ◽  
Igor Linkov
Keyword(s):  
Synthetic Biology ◽  
Genetic Engineering ◽  
Biomedical Research ◽  
Gene Editing ◽  
Basic Research ◽  
Eukaryotic Cells ◽  
New Developments ◽  
Standards Of Living ◽  
New Challenges ◽  
Almost All

AbstractNovel biology technologies like gene editing and genetic engineering are creating a proliferation of breakthroughs in engineered biological systems that will change our world in areas ranging from medicine, to textiles, to energy. New developments in gene editing technologies, especially CRISPR-Cas9, have shown early signs of extraordinary potential in a variety of fields, including from basic research, applied biotechnology, and biomedical research. While the possibility of directly targeting and modifying genomic sequences in almost all eukaryotic cells could significantly improve standards of living, these technologies have the potential to pose serious biological hazards.

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