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 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 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.

Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases

2019 ◽  
Vol 88 (1) ◽  
pp. 191-220 ◽  
Author(s):  
Daesik Kim ◽  
Kevin Luk ◽  
Scot A. Wolfe ◽  
Jin-Soo Kim
Keyword(s):  
Dna Sequences ◽  
Gene Editing ◽  
Genomic Rearrangements ◽  
Zinc Finger Nucleases ◽  
Collateral Damage ◽  
Transcription Activator ◽  
Genome Wide ◽  
Target Sites ◽  
Programmable Nucleases ◽  
Target Activity

Programmable nucleases and deaminases, which include zinc-finger nucleases, transcription activator-like effector nucleases, CRISPR RNA-guided nucleases, and RNA-guided base editors, are now widely employed for the targeted modification of genomes in cells and organisms. These gene-editing tools hold tremendous promise for therapeutic applications. Importantly, these nucleases and deaminases may display off-target activity through the recognition of near-cognate DNA sequences to their target sites, resulting in collateral damage to the genome in the form of local mutagenesis or genomic rearrangements. For therapeutic genome-editing applications with these classes of programmable enzymes, it is essential to measure and limit genome-wide off-target activity. Herein, we discuss the key determinants of off-target activity for these systems. We describe various cell-based and cell-free methods for identifying genome-wide off-target sites and diverse strategies that have been developed for reducing the off-target activity of programmable gene-editing enzymes.

scholarly journals Improving Animal Diets to Increase Relevance to Human Populations

2017 ◽  
Vol 7 (5) ◽  
pp. 329
Author(s):  
Korry J. Hintze ◽  
Abby D. Benninghoff ◽  
Robert E. Ward
Keyword(s):  
Animal Models ◽  
Biomedical Research ◽  
Allometric Scaling ◽  
Human Populations ◽  
Rodent Models ◽  
Nutrient Density ◽  
Laboratory Rodent ◽  
Research Findings ◽  
Over Time

Background: Rodent models have been an invaluable resource for biomedical research and have been instrumental for countless advances in our understanding of biology and human disease.  However, inherent to using these models is the issue of translatability of research findings to human populations. Some differences between humans and rodents can never be reconciled because of key differences in physiology.  However, rodent models have evolved over time through innovations in genetics and standardized animal diets, resulting in reduced variability across experiments. Developing animal diets that more closely emulate what humans eat will help increase the translational fidelity of animal models to human populations. This review will focus on the role of basal laboratory diets for improving animal models. Keywords: laboratory rodent diets, total Western Diet, allometric scaling, nutrient density scaling 

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.

scholarly journals Mitochondria in Health and Diseases

Cells ◽  
2020 ◽  
Vol 9 (5) ◽  
pp. 1177 ◽  
Author(s):  
Sabzali Javadov ◽  
Andrey V. Kozlov ◽  
Amadou K. S. Camara
Keyword(s):  
Cell Death ◽  
Animal Models ◽  
Human Diseases ◽  
Eukaryotic Cells ◽  
Special Issue ◽  
Subcellular Organelles ◽  
The Past ◽  
Cell Life ◽  
And Function

Mitochondria are subcellular organelles evolved by endosymbiosis of bacteria with eukaryotic cells characteristics. They are the main source of ATP in the cell and play a pivotal role in cell life and cell death. Mitochondria are engaged in the pathogenesis of human diseases and aging directly or indirectly through a broad range of signaling pathways. However, despite an increased interest in mitochondria over the past decades, the mechanisms of mitochondria-mediated cell/organ dysfunction in response to pathological stimuli remain unknown. The Special Issue, “Mitochondria in Health and Diseases,” organized by Cells includes 24 review and original articles that highlight the latest achievements in elucidating the role of mitochondria under physiological (healthy) conditions and, in various cell/animal models of human diseases and, in patients. Altogether, the Special Issue summarizes and discusses different aspects of mitochondrial metabolism and function that open new avenues in understanding mitochondrial biology.

Cellular and animal models in pheochromocytoma/paragangliomas research: role of microenvironment

2016 ◽  
Author(s):  
M Mannelli ◽  
E Rapizzi ◽  
L Canu ◽  
T Ercolino ◽  
V Giache
Keyword(s):  
Animal Models

Quality assurance and quality improvement in medical practice – Part 1. Definition and importance of quality in medical practice

2012 ◽  
Vol 153 (3) ◽  
pp. 83-92
Author(s):  
Sándor Gődény
Keyword(s):  
Quality Assurance ◽  
Health Status ◽  
Quality Management ◽  
Medical Practice ◽  
Quality Measurement ◽  
The European Union ◽  
Management Quality ◽  
Hungarian Population ◽  
Healthcare Finance

In Hungary healthcare finance has decreased in proportion with the GDP, while the health status of the population is still ranks among the worst in the European Union. Since healthcare finance is not expected to increase, the number of practicing doctors per capita is continuously decreasing. In the coming years it is an important question that in this situation what methods can be used to prevent further deterioration of the health status of the Hungarian population, and within this is the role of the quality approach, and different methods of quality management. In the present and the forthcoming two articles those standpoints will be summarized which support the need for the integration of quality assurance in the everyday medical practice. In the first part the importance of quality thinking, quality management, quality assurance, necessity of quality measurement and improvement, furthermore, advantages of the quality systems will be discussed. Orv. Hetil., 2012, 153, 83–92.

Bioengineered in vitro Vascular Models for Applications in Interventional Radiology

2019 ◽  
Vol 24 (45) ◽  
pp. 5367-5374 ◽  
Author(s):  
Xiaoyun Li ◽  
Seyed M. Moosavi-Basri ◽  
Rahul Sheth ◽  
Xiaoying Wang ◽  
Yu S. Zhang
Keyword(s):  
Interventional Radiology ◽  
Animal Models ◽  
Blood Vessels ◽  
In Vitro Models ◽  
The Past ◽  
Endovascular Interventions ◽  
Conventional Animal ◽  
Vascular Structures

The role of endovascular interventions has progressed rapidly over the past several decades. While animal models have long-served as the mainstay for the advancement of this field, the use of in vitro models has become increasingly widely adopted with recent advances in engineering technologies. Here, we review the strategies, mainly including bioprinting and microfabrication, which allow for fabrication of biomimetic vascular models that will potentially serve to supplement the conventional animal models for convenient investigations of endovascular interventions. Besides normal blood vessels, those in diseased states, such as thrombosis, may also be modeled by integrating cues that simulate the microenvironment of vascular disorders. These novel engineering strategies for the development of biomimetic in vitro vascular structures will possibly enable unconventional means of studying complex endovascular intervention problems that are otherwise hard to address using existing models.

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