scholarly journals Gene editing in dermatology: Harnessing CRISPR for the treatment of cutaneous disease

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 281
Author(s):  
Catherine Baker ◽  
Matthew S. Hayden
Keyword(s):  
Human Genome ◽  
Genome Editing ◽  
Gene Editing ◽  
Human Diseases ◽  
Therapeutic Application ◽  
Cutaneous Disease ◽  
Crispr System ◽  
Novel Approaches

The discovery of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system has revolutionized gene editing research. Through the repurposing of programmable RNA-guided CRISPR-associated (Cas) nucleases, CRISPR-based genome editing systems allow for the precise modification of specific sites in the human genome and inspire novel approaches for the study and treatment of inherited and acquired human diseases. Here, we review how CRISPR technologies have stimulated key advances in dermatologic research.  We discuss the role of CRISPR in genome editing for cutaneous disease and highlight studies on the use of CRISPR-Cas technologies for genodermatoses, cutaneous viruses and bacteria, and melanoma. Additionally, we examine key limitations of current CRISPR technologies, including the challenges these limitations pose for the widespread therapeutic application of CRISPR-based therapeutics.

scholarly journals Gene editing in dermatology: Harnessing CRISPR for the treatment of cutaneous disease

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 281
Author(s):  
Catherine Baker ◽  
Matthew S. Hayden
Keyword(s):  
Human Genome ◽  
Genome Editing ◽  
Gene Editing ◽  
Human Diseases ◽  
Therapeutic Application ◽  
Cutaneous Disease ◽  
Crispr System ◽  
Novel Approaches

The discovery of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system has revolutionized gene editing research. Through the repurposing of programmable RNA-guided CRISPR-associated (Cas) nucleases, CRISPR-based genome editing systems allow for the precise modification of specific sites in the human genome and inspire novel approaches for the study and treatment of inherited and acquired human diseases. Here, we review how CRISPR technologies have stimulated key advances in dermatologic research.  We discuss the role of CRISPR in genome editing for cutaneous disease and highlight studies on the use of CRISPR-Cas technologies for genodermatoses, cutaneous viruses and bacteria, and melanoma. Additionally, we examine key limitations of current CRISPR technologies, including the challenges these limitations pose for the widespread therapeutic application of CRISPR-based therapeutics.

Regulating genome editing technologies: a comparison of expert recommendations in the U.K. and in the U.S.A.

2019 ◽  
Vol 3 (6) ◽  
pp. 701-705
Author(s):  
Sofia Iacomussi
Keyword(s):  
Human Genome ◽  
Genome Editing ◽  
Public Engagement ◽  
General Public ◽  
Regulatory Framework ◽  
Socially Responsible ◽  
Scientific Experts ◽  
Bioethical Debate ◽  
Expert Recommendations

The present paper aims to inform the bioethical debate on the regulation of human genome editing technologies with a specific focus on the role of scientific experts and their interactions with the general public in the formulation of policy. It reviews and compares two of the major contributions to this debate in the U.K. and in the U.S.A., comparing expert approaches towards regulation on genome editing technologies. The results of this analysis offer important lessons that should be appreciated in building an international regulatory framework. On the basis of these results, I conclude that the experts should embrace a socially responsible approach and encourage active public engagement.

Advances in therapeutic application of CRISPR-Cas9

2019 ◽  
Vol 19 (3) ◽  
pp. 164-174 ◽  
Author(s):  
Jinyu Sun ◽  
Jianchu Wang ◽  
Donghui Zheng ◽  
Xiaorong Hu
Keyword(s):  
Gene Therapy ◽  
Genome Editing ◽  
Malignant Tumor ◽  
Gene Editing ◽  
Genome Engineering ◽  
Therapeutic Application ◽  
Adaptive Immune ◽  
Efficient Gene

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) is one of the most versatile and efficient gene editing technologies, which is derived from adaptive immune strategies for bacteria and archaea. With the remarkable development of programmable nuclease-based genome engineering these years, CRISPR-Cas9 system has developed quickly in recent 5 years and has been widely applied in countless areas, including genome editing, gene function investigation and gene therapy both in vitro and in vivo. In this paper, we briefly introduce the mechanisms of CRISPR-Cas9 tool in genome editing. More importantly, we review the recent therapeutic application of CRISPR-Cas9 in various diseases, including hematologic diseases, infectious diseases and malignant tumor. Finally, we discuss the current challenges and consider thoughtfully what advances are required in order to further develop the therapeutic application of CRISPR-Cas9 in the future.

The Ethics of Human Tripronuclear Zygotes as Germline Editing Subjects

2019 ◽  
Vol 19 (3) ◽  
pp. 429-442
Author(s):  
Jeanatan Hall ◽  
Keyword(s):  
Human Genome ◽  
Genome Editing ◽  
Gene Editing ◽  
Medical Waste ◽  
Compelling Evidence ◽  
Acceptable Alternative ◽  
Development Cycle ◽  
Genetic Abnormalities ◽  
Normal Human ◽  
Human Zygotes

Despite great interest in the field of gene editing, sparked by the advent of CRISPR/Cas9-mediated applications, the personhood of tripronuclear zygotes has not been addressed appropriately. 3PN zygotes are discarded as medical waste, and their use as models for human genome editing is becoming increasing common. 3PN zygotes possess an extra set of chromosomes, which often leads to severe genetic abnormalities; they are dismissed as “nonviable embryos” and treated as an ethically acceptable alternative to human embryonic research. However, given the development cycle of 3PN zygotes and the qualifications for human personhood assessed, there is compelling evidence that 3PN zygotes are indeed human persons. Although genetically disadvantaged, they deserve the same respect as do genetically normal human zygotes.

scholarly journals CRISPR Pioneers Win 2020 Nobel Prize for Chemistry

2020 ◽  
Author(s):  
Dariush D. FARHUD ◽  
Marjan ZARIF-YEGANEH
Keyword(s):  
Clinical Practice ◽  
Human Genome ◽  
Genome Editing ◽  
Nobel Prize ◽  
Gene Editing ◽  
High Accuracy ◽  
Recent Advances ◽  
Programmable Nucleases

Over the last few years, the development of genome editing has revolutionized research on the human genome. Recent advances in developing programmable nucleases, such as meganucleases, ZFNs, TALENs and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas, has greatly expedited the progress of gene editing from concept to clinical practice. The CRISPR has advantages over other nuclease-based genome editing tools due to its high accuracy, efficiency, and strong specificity. Eight years after CRISPR application for human genome edition by Emmanuelle Charpentier and Jennifer A. Doudna, the 2020 Nobel Prize in Chemistry has been jointly given to them for development of CRISPR-Cas9 gene editing, allows scientists to precisely cut and edit of DNA.

scholarly journals ROLE OF CRISPR TO IMPROVE ABIOTIC STRESS TOLERANCE IN CROP PLANTS

2021 ◽  
Vol 2021 (1) ◽  
Author(s):  
MU Farooq ◽  
MF Bashir ◽  
MUS Khan ◽  
B Iqbal ◽  
Q Ali
Keyword(s):  
Abiotic Stress ◽  
Genome Editing ◽  
Gene Editing ◽  
Abiotic Stress Tolerance ◽  
Genetically Engineered ◽  
Transcription Activator ◽  
Guide Rnas ◽  
Field Crops ◽  
Genetically Engineered Crops

The study for genetic variation in plant genomes for a variety of crops, as well as developments of genome editing techniques, have made it possible to cultivate for about any desired trait. Zinc finger enzymes; have made strides in genome-editing. Molecular biologists can now more specifically target every gene using transcription activator-like effector nucleases and ZFNs. These methods, on the other hand, are expensive and time-consuming because they involve complex procedures. Referring to various genome editing techniques, CRISPR/Cas9 genetic modification is simple to construct and clone and the Cas9 could be used with different guide RNAs controlling different genes. Following solid evidence demonstrations using the main CRISPR-Cas9 unit in field crops, multiple updated Cas9 cassettes are often used in plant species to improve target precision and reduce off target cleavage. Nmcas9, Sacas9, as well as Stcas9 are a few examples. Furthermore, Cas9 enzymes are readily available from a variety of sources. Bacteria that had never been discovered before has found solutions available to improve specificity and efficacy of gene editing techniques. The choices are summarized in this analysis to plant's experiment to develop crops using CRISPR/Cas9 technology; the tolerance of biotic & abiotic stress may be improved. These strategies will lead to the growth of non-genetically engineered crops with the target phenotype, which will further improve yield capacity under biotic & abiotic stress environments.

scholarly journals Potential of Mitochondrial Genome Editing for Human Fertility Health

2021 ◽  
Vol 12 ◽  
Author(s):  
Lin Fu ◽  
Yu-Xin Luo ◽  
Ying Liu ◽  
Hui Liu ◽  
Hong-zhen Li ◽  
...  
Keyword(s):  
Mitochondrial Genome ◽  
Genome Editing ◽  
Gene Editing ◽  
Rapid Development ◽  
Normal Functioning ◽  
Complex Processes ◽  
Human Fertility ◽  
Fertility Disorders ◽  
Made In

Mitochondrial DNA (mtDNA) encodes vital proteins and RNAs for the normal functioning of the mitochondria. Mutations in mtDNA leading to mitochondrial dysfunction are relevant to a large spectrum of diseases, including fertility disorders. Since mtDNA undergoes rather complex processes during gametogenesis and fertilization, clarification of the changes and functions of mtDNA and its essential impact on gamete quality and fertility during this process is of great significance. Thanks to the emergence and rapid development of gene editing technology, breakthroughs have been made in mitochondrial genome editing (MGE), offering great potential for the treatment of mtDNA-related diseases. In this review, we summarize the features of mitochondria and their unique genome, emphasizing their inheritance patterns; illustrate the role of mtDNA in gametogenesis and fertilization; and discuss potential therapies based on MGE as well as the outlook in this field.

scholarly journals Modeling Neuronal Diseases in Zebrafish in the Era of CRISPR

2020 ◽  
Vol 18 (2) ◽  
pp. 136-152
Author(s):  
Angeles Edith Espino-Saldaña ◽  
Roberto Rodríguez-Ortiz ◽  
Elizabeth Pereida-Jaramillo ◽  
Ataúlfo Martínez-Torres
Keyword(s):  
Brain Function ◽  
Rapid Development ◽  
Living Organism ◽  
Human Diseases ◽  
Brain Diseases ◽  
Neuronal Function ◽  
Brain Organization ◽  
Crispr System ◽  
Technology Applications

Background: Danio rerio is a powerful experimental model for studies in genetics and development. Recently, CRISPR technology has been applied in this species to mimic various human diseases, including those affecting the nervous system. Zebrafish offer multiple experimental advantages: external embryogenesis, rapid development, transparent embryos, short life cycle, and basic neurobiological processes shared with humans. This animal model, together with the CRISPR system, emerging imaging technologies, and novel behavioral approaches, lay the basis for a prominent future in neuropathology and will undoubtedly accelerate our understanding of brain function and its disorders. Objective: Gather relevant findings from studies that have used CRISPR technologies in zebrafish to explore basic neuronal function and model human diseases. Method: We systematically reviewed the most recent literature about CRISPR technology applications for understanding brain function and neurological disorders in D. rerio. We highlighted the key role of CRISPR in driving forward our understanding of particular topics in neuroscience. Results: We show specific advances in neurobiology when the CRISPR system has been applied in zebrafish and describe how CRISPR is accelerating our understanding of brain organization. Conclusion: Today, CRISPR is the preferred method to modify genomes of practically any living organism. Despite the rapid development of CRISPR technologies to generate disease models in zebrafish, more efforts are needed to efficiently combine different disciplines to find the etiology and treatments for many brain diseases.

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 Multiplex gene editing by CRISPR-Cpf1 through autonomous processing of a single crRNA array

2016 ◽  
Author(s):  
Bernd Zetsche ◽  
Matthias Heidenreich ◽  
Prarthana Mohanraju ◽  
Iana Fedorova ◽  
Jeroen Kneppers ◽  
...  
Keyword(s):  
Genome Editing ◽  
Mouse Brain ◽  
Mammalian Cells ◽  
Gene Editing ◽  
Simple Route ◽  
Defense Systems ◽  
Crispr System ◽  
Crispr Array ◽  
Type V

Microbial CRISPR-Cas defense systems have been adapted as a platform for genome editing applications built around the RNA-guided effector nucleases, such as Cas9. We recently reported the characterization of Cpf1, the effector nuclease of a novel type V-A CRISPR system, and demonstrated that it can be adapted for genome editing in mammalian cells (Zetsche et al., 2015). Unlike Cas9, which utilizes a trans-activating crRNA (tracrRNA) as well as the endogenous RNaseIII for maturation of its dual crRNA:tracrRNA guides (Deltcheva et al., 2011), guide processing of the Cpf1 system proceeds in the absence of tracrRNA or other Cas (CRISPR associated) genes (Zetsche et al., 2015) (Figure 1a), suggesting that Cpf1 is sufficient for pre-crRNA maturation. This has important implications for genome editing, as it would provide a simple route to multiplex targeting. Here, we show for two Cpf1 orthologs that no other factors are required for array processing and demonstrate multiplex gene editing in mammalian cells as well as in the mouse brain by using a designed single CRISPR array.

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