A critical view of CRISPR applications in Human Genetics

2020 ◽  
Vol 1 (1) ◽  
Author(s):  
José Luis Ramírez Ochoa
Keyword(s):  
Gene Therapy ◽  
Human Health ◽  
Gene Editing ◽  
Human Genetics ◽  
Comprehensive Review ◽  
New Era ◽  
Critical View ◽  
New Horizons ◽  
Ethical Concerns ◽  
Short Period

Gene therapy entered a new era with CRISPR gene-editing technology. In a short period of eight years, CRISPR revolutionized how we handle genes by simplifying and democratizing the tools for gene editing. CRISPR applications in human health are exponentially growing and opening new horizons for otherwise difficult diseases to treat. Nonetheless, the versatility, easiness, and wide use of this technology in humans are creating new bioethical challenges. Here, aiming to non-experts, students, and researchers, I briefly review some relevant aspects of CRISPR history and evolution, as well as some ethical concerns related to its use in humans. To pretend to do a comprehensive review on CRISPR is a futile task since the speed of its evolution would render it obsolete by the time of its publication.

scholarly journals Gene Therapy for Cardiovascular Disease: Basic Research and Clinical Prospects

2021 ◽  
Vol 8 ◽  
Author(s):  
Genmao Cao ◽  
Xuezhen Xuan ◽  
Ruijing Zhang ◽  
Jie Hu ◽  
Honglin Dong
Keyword(s):  
Cardiovascular Disease ◽  
Gene Therapy ◽  
Cardiovascular Diseases ◽  
Gene Editing ◽  
Rapid Development ◽  
Basic Research ◽  
Early Years ◽  
Transcription Activator ◽  
Term Survival ◽  
Ethical Concerns

In recent years, the vital role of genetic factors in human diseases have been widely recognized by scholars with the deepening of life science research, accompanied by the rapid development of gene-editing technology. In early years, scientists used homologous recombination technology to establish gene knock-out and gene knock-in animal models, and then appeared the second-generation gene-editing technology zinc-finger nucleases (ZFNs) and transcription activator–like effector nucleases (TALENs) that relied on nucleic acid binding proteins and endonucleases and the third-generation gene-editing technology that functioned through protein–nucleic acids complexes—CRISPR/Cas9 system. This holds another promise for refractory diseases and genetic diseases. Cardiovascular disease (CVD) has always been the focus of clinical and basic research because of its high incidence and high disability rate, which seriously affects the long-term survival and quality of life of patients. Because some inherited cardiovascular diseases do not respond well to drug and surgical treatment, researchers are trying to use rapidly developing genetic techniques to develop initial attempts. However, significant obstacles to clinical application of gene therapy still exists, such as insufficient understanding of the nature of cardiovascular disease, limitations of genetic technology, or ethical concerns. This review mainly introduces the types and mechanisms of gene-editing techniques, ethical concerns of gene therapy, the application of gene therapy in atherosclerosis and inheritable cardiovascular diseases, in-stent restenosis, and delivering systems.

scholarly journals Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases

2021 ◽  
Vol 22 (24) ◽  
pp. 13674
Author(s):  
Giulia Paolini Sguazzi ◽  
Valentina Muto ◽  
Marco Tartaglia ◽  
Enrico Bertini ◽  
Claudia Compagnucci
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Stem Cell ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Cell Biology ◽  
Recent Progress ◽  
Gene Editing ◽  
New Era ◽  
Induced Pluripotent

To date, gene therapy has employed viral vectors to deliver therapeutic genes. However, recent progress in molecular and cell biology has revolutionized the field of stem cells and gene therapy. A few years ago, clinical trials started using stem cell replacement therapy, and the induced pluripotent stem cells (iPSCs) technology combined with CRISPR-Cas9 gene editing has launched a new era in gene therapy for the treatment of neurological disorders. Here, we summarize the latest findings in this research field and discuss their clinical applications, emphasizing the relevance of recent studies in the development of innovative stem cell and gene editing therapeutic approaches. Even though tumorigenicity and immunogenicity are existing hurdles, we report how recent progress has tackled them, making engineered stem cell transplantation therapy a realistic option.

scholarly journals Latest Advances of Virology Research Using CRISPR/Cas9-Based Gene-Editing Technology and Its Application to Vaccine Development

Viruses ◽  
2021 ◽  
Vol 13 (5) ◽  
pp. 779
Author(s):  
Man Teng ◽  
Yongxiu Yao ◽  
Venugopal Nair ◽  
Jun Luo
Keyword(s):  
Biological Sciences ◽  
Gene Therapy ◽  
Genetic Engineering ◽  
Gene Function ◽  
Viral Genome ◽  
Gene Editing ◽  
Vaccine Development ◽  
Future Prospects ◽  
Dna Viruses ◽  
Viral Genomes

In recent years, the CRISPR/Cas9-based gene-editing techniques have been well developed and applied widely in several aspects of research in the biological sciences, in many species, including humans, animals, plants, and even in viruses. Modification of the viral genome is crucial for revealing gene function, virus pathogenesis, gene therapy, genetic engineering, and vaccine development. Herein, we have provided a brief review of the different technologies for the modification of the viral genomes. Particularly, we have focused on the recently developed CRISPR/Cas9-based gene-editing system, detailing its origin, functional principles, and touching on its latest achievements in virology research and applications in vaccine development, especially in large DNA viruses of humans and animals. Future prospects of CRISPR/Cas9-based gene-editing technology in virology research, including the potential shortcomings, are also discussed.

Comprehensive review on lactate metabolism in human health

Mitochondrion ◽  
2014 ◽  
Vol 17 ◽  
pp. 76-100 ◽  
Author(s):  
M. Adeva-Andany ◽  
M. López-Ojén ◽  
R. Funcasta-Calderón ◽  
E. Ameneiros-Rodríguez ◽  
C. Donapetry-García ◽  
...  
Keyword(s):  
Human Health ◽  
Lactate Metabolism ◽  
Comprehensive Review

Potential HIV gene therapy strategies

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Cord Blood ◽  
Gene Editing ◽  
Mortality Risks ◽  
Recent Advances ◽  
Whole Exome ◽  
Localization Signal ◽  
Cord Blood Cells ◽  
The Cost ◽  
Therapy Strategies

The CCR5 null genotype generation has been a main focus in the HIV gene therapy industry. The presence of the X4 tropic virus, mobilization of HSPCs, the quality of the cells for manipulation, and gene editing efficiency appear to be the main obstacles in translating this technique. Unintended off-target cleavage is a key problem in CRISPR/Cas9 editing. With the development of small molecule expansion methods for cord blood HSPC, it would be advantageous to modify CCR5 in cord blood cells and expand them for transplantation. The generation of engraftable HSPCS from iPSCs would be an ideal technique for HSCC gene therapy.The haplotype-characterized iPSC would be the donor for many patients, and it could be a commercially available product. The 32 C CR5 homozygous people had no elevated mortality risks according to whole-exome sequencing and whole-genome genotyping, according to CCR 5 positive people, and had no higher mortality risks compared to those who were HIV positive. Recent advances in gene editing, such as non-viral delivery of Cas9 ribonucleoproteins, incorporation of a 3X-nuclear localization signal into spCas9, and use of HiFi Cas9 with chemically modified sgRNAs, can be combined with recent advances in transplantation. Infusing modest doses of gene modified primitive HSPC fractions indicated by CD34 + CD90 + CD45RA-, which can engraft better, is another option for lowering the cost of gene therapy.

scholarly journals Gene editing using CRISPR-Cas9 for the treatment of lung cancer

2016 ◽  
pp. 178-180 ◽  
Author(s):  
Andres Castillo
Keyword(s):  
Applied Sciences ◽  
Lung Cancer ◽  
High Precision ◽  
Genetic Information ◽  
Gene Editing ◽  
Therapeutic Strategies ◽  
Technological Advance ◽  
Genetic Origin ◽  
New Era ◽  
Health Area

In 2015, the journal Science chose CRISPR-Cas9 technology as the most important technological advance of science in the last years. This magazine announced the beginning of a new era of biotechnology in which it would be possible to edit, correct and modify the genetic information of any cell in a feasible, fast and cheap way; and most importantly, with high precision. Its implementation in research laboratories in basic and applied sciences could help to develop therapeutic strategies for the health area with the main objective of healing diseases with a known genetic origin, and that until now have been impossible to cure

scholarly journals New Horizons: Graduate Music Therapy Studies in Québec, Canada

2011 ◽  
Vol 11 (3) ◽  
Author(s):  
Sandi Curtis
Keyword(s):  
Music Therapy ◽  
New Era ◽  
New Horizons ◽  
Therapy Program ◽  
Concordia University ◽  
Therapy Studies ◽  
The City

The year 2011 marks the start of a new era for music therapy in the province of Québec with the official launch of its first and only graduate music therapy program. This program, situated at Concordia University in the heart of the city of Montréal, is described, along with its development, its purpose, its role in the changing landscape of music therapy in Québec, and its defining characteristics. Future ramifications for music therapy at Concordia University and in Québec and the rest of Canada are outlined.

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