Development Of Novel Crispr Technologies For The Precise Genome Editing Of Human Pluripotent Stem Cells PDF Download
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Author | : Jared Matthew Carlson-Stevermer |
Publisher | : |
Total Pages | : 0 |
Release | : 2018 |
Genre | : |
ISBN | : |
Download Development of Novel CRISPR Technologies for the Precise Genome Editing of Human Pluripotent Stem Cells Book in PDF, ePub and Kindle
Precision genome surgery has the potential to revolutionize the field of personalized medicine through applications ranging from basic science, to creation of in vitro disease models, all the way to the correction of genetic disease in vivo. Although the field has rapidly advanced since the development of CRISPR-based technology, the ability to generate precise edits at multiple locations within the genome remains an outstanding challenge. As a result, edited cell populations as well as isolated clones must be rigorously screened for the presence of undesired mutations before any form of clinical application such as ex vivo cell therapy or in vivo somatic cell editing can be undertaken. Thus, there is a need to increase rates of precise editing and decrease rates of imprecise mutation to improve the clinical viability of gene editing. This thesis aims to develop new technologies to address challenges in precision genome editing and subsequently demonstrate their utility in a human pluripotent stem cell disease model. First, I describe a method to track hundreds of cell populations in real time using high-content analysis, allowing the facile selection of those that have undergone genome editing. Second, I demonstrate a new technology that is capable of tethering together CRISPR ribonucleoproteins and donor DNA to increase the frequency of precise gene surgery. Finally, I combine these technologies to correct mutations in an induced pluripotent stem cell model of Pompe disease, an autosomal recessive disorder. These methods and their applications demonstrate advances in the field of precision genome surgery and represent an important step toward the clinical use of genetic therapies.
Author | : The Royal Society |
Publisher | : National Academies Press |
Total Pages | : 239 |
Release | : 2021-01-16 |
Genre | : Medical |
ISBN | : 0309671132 |
Download Heritable Human Genome Editing Book in PDF, ePub and Kindle
Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight.
Author | : Ziheng Zhang |
Publisher | : Springer Nature |
Total Pages | : 245 |
Release | : 2022-02-21 |
Genre | : Science |
ISBN | : 9811685045 |
Download CRISPR Book in PDF, ePub and Kindle
The book presents a machine-generated literature review on CRISPR (clustered regularly interspaced short palindromic repeats) from 114 selected papers published by Springer Nature in the last few years, which are then organized by the book editors with a human-written introduction to each chapter. Each chapter presents summaries of predefined themes and provides the reader with a basis for further exploration of the topic. As one of the experimental projects initiated by Springer Nature for AI book content generation, this book shows the latest developments in the CRISPR field. It will be a useful reference for graduate students who are interested in CRISPR-related research and early-career researchers who need an overview of the current development of the field.
Author | : Stephen H. Tsang |
Publisher | : Springer |
Total Pages | : 180 |
Release | : 2017-11-11 |
Genre | : Science |
ISBN | : 3319639048 |
Download Precision Medicine, CRISPR, and Genome Engineering Book in PDF, ePub and Kindle
This book presents descriptive overviews of gene editing strategies across multiple species while also offering in-depth insight on complex cases of application in the field of tissue engineering and regenerative medicine. Chapters feature contributions from leaders in stem cell therapy and biology, providing a comprehensive view of the application of gene therapy in numerous fields with an emphasis on ophthalmology, stem cells, and agriculture. The book also highlights recent major technological advances, including ZFN, TALEN, and CRISPR. Precision Medicine, CRISPR, and Genome Engineering is part of the highly successful Advances in Experimental Medicine and Biology series. It is an indispensable resource for researchers and students in genetics as well as clinicians.
Author | : Rudolf Jaenisch |
Publisher | : Springer |
Total Pages | : 129 |
Release | : 2017-09-14 |
Genre | : Medical |
ISBN | : 331960192X |
Download Genome Editing in Neurosciences Book in PDF, ePub and Kindle
This book is open access under a CC BY 4.0 license. CRISPR-Cas9 is a rapid, efficient, versatile and relatively cheap method for dissecting the molecular pathways that are the basis of life, as well as for investigating and potentially rectifying faults in these pathways that result in disease. This book reviews how CRISPR-Cas9 and other genome editing techniques are advancing our understanding of development and function in the nervous system, uncovering the molecular causes of neurological disorders and providing tools for gene therapy.
Author | : Rodolphe Barrangou |
Publisher | : Springer Science & Business Media |
Total Pages | : 300 |
Release | : 2012-12-13 |
Genre | : Science |
ISBN | : 364234657X |
Download CRISPR-Cas Systems Book in PDF, ePub and Kindle
CRISPR/Cas is a recently described defense system that protects bacteria and archaea against invasion by mobile genetic elements such as viruses and plasmids. A wide spectrum of distinct CRISPR/Cas systems has been identified in at least half of the available prokaryotic genomes. On-going structural and functional analyses have resulted in a far greater insight into the functions and possible applications of these systems, although many secrets remain to be discovered. In this book, experts summarize the state of the art in this exciting field.
Author | : Stephen H. Tsang |
Publisher | : Academic Press |
Total Pages | : 138 |
Release | : 2021-10-20 |
Genre | : Science |
ISBN | : 0128178779 |
Download CRISPR Genome Surgery in Stem Cells and Disease Tissues Book in PDF, ePub and Kindle
CRISPR Genome Surgery in Stem Cells and Disease Tissues focuses uniquely on the clinical applications of CRISPR/Cas9 based technology. Topics include the latest advances in gene editing and its translational applications to various diseases, including retinal degenerative disease, recessively inherited diseases, and dominantly inherited diseases, to name a few. The book's target audience includes researchers, students, clinicians and the general public. This space that is not currently served by any existing resource, so this publication fills a gap in current literature. Provides a thorough review of CRISPR-Cas9, from discovery to therapy Covers the latest advances in gene editing and its translational applications to various diseases Written by global leaders in the fields of gene editing and stem cell therapy
Author | : Rudolf Jaenisch |
Publisher | : |
Total Pages | : 128 |
Release | : 2020-10-08 |
Genre | : Medical |
ISBN | : 9781013268564 |
Download Genome Editing in Neurosciences Book in PDF, ePub and Kindle
Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors.
Author | : National Academies of Sciences, Engineering, and Medicine |
Publisher | : National Academies Press |
Total Pages | : 329 |
Release | : 2017-08-13 |
Genre | : Medical |
ISBN | : 0309452880 |
Download Human Genome Editing Book in PDF, ePub and Kindle
Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Author | : Zhiying Zhang |
Publisher | : Frontiers Media SA |
Total Pages | : 135 |
Release | : 2020-06-04 |
Genre | : |
ISBN | : 2889637794 |
Download Precise Genome Editing Techniques and Applications Book in PDF, ePub and Kindle