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CRISPR Genome Surgery in Stem Cells and Disease Tissues

CRISPR Genome Surgery in Stem Cells and Disease Tissues
Author: Stephen H. Tsang
Publisher: Academic Press
Total Pages: 138
Release: 2021-10-20
Genre: Science
ISBN: 0128178779

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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


CRISPR: Genome Editing and Engineering And Related Issues

CRISPR: Genome Editing and Engineering And Related Issues
Author: Barbara Wexler
Publisher: Gale, Cengage Learning
Total Pages: 11
Release: 2018-11-16
Genre: Science
ISBN: 0028666682

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eBook content that offers a clear and comprehensive introduction to CRISPR and related topics. Entries include foundational concepts, key scientific figures and historical themes, ethical issues , and advances in the science.


Precision Medicine, CRISPR, and Genome Engineering

Precision Medicine, CRISPR, and Genome Engineering
Author: Stephen H. Tsang
Publisher: Springer
Total Pages: 180
Release: 2017-11-11
Genre: Science
ISBN: 3319639048

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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.


CRISPR-/Cas9 Based Genome Editing for Treating Genetic Disorders and Diseases

CRISPR-/Cas9 Based Genome Editing for Treating Genetic Disorders and Diseases
Author: Luis María Vaschetto
Publisher: CRC Press
Total Pages: 268
Release: 2022-01-31
Genre: Mathematics
ISBN: 1000540812

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The CRISPR-Cas9 genome-editing system is creating a revolution in the science world. In the laboratory, CRISPR-Cas9 can efficiently be used to target specific genes, correct mutations and regulate gene expression of a wide array of cells and organisms, including human cells. CRISPR-/Cas9 Based Genome Editing for Treating Genetic Disorders and Diseases is a unique reading material for college students, academicians, and other health professionals interested in learning about the broad range of applications of CRISPR/Cas9 genetic scissors. Some topics included in this book are: the role of the CRISPR/Cas9 system in neuroscience, gene therapy, epigenome editing, genome mapping, cancer, virus infection control strategies, regulatory challenges and bioethical considerations.


Development of Novel CRISPR Technologies for the Precise Genome Editing of Human Pluripotent Stem Cells

Development of Novel CRISPR Technologies for the Precise Genome Editing of Human Pluripotent Stem Cells
Author: Jared Matthew Carlson-Stevermer
Publisher:
Total Pages: 0
Release: 2018
Genre:
ISBN:

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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.


Stem Cells for Cancer and Genetic Disease Treatment

Stem Cells for Cancer and Genetic Disease Treatment
Author: Phuc Van Pham
Publisher: Springer
Total Pages: 190
Release: 2018-11-07
Genre: Science
ISBN: 3319980653

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This invaluable resource discusses insights ranging from basic biological mechanisms of various types of stem cells through the potential applications in the treatment of human diseases, including cancer and genetic disorders. These discoveries are placed within the structural context of tissue and developmental biology in sections dealing with recent advances in understanding different types of stem cell biology and their potential applications in tissue repair and regeneration and in the treatment different types of human cancer and genetic diseases or disorders. Stem Cells for Cancer and Genetic Disease Treatment and the other books in the Stem Cells in Clinical Applicationsseries will be invaluable to scientists, researchers, advanced students and clinicians working in stem cells, regenerative medicine or tissue engineering as well as cancer or genetics research.


Autologous And Cancer Stem Cell Gene Therapy

Autologous And Cancer Stem Cell Gene Therapy
Author: Roger Bertolotti
Publisher: World Scientific
Total Pages: 489
Release: 2007-12-13
Genre: Medical
ISBN: 9814472557

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Stem cells provide for life-long cell replacement in tissues and organs, and have inherent homing abilities that are critical in therapeutic applications. Stem cells are also the driving force of cancer where genetic/epigenetic alterations culminate in tumorigenesis either in tissue stem cells or in some of their derivatives. As a rare subset of the tumor, cancer stem cells are the only drive of tumor initiation/propagation. Autologous and cancer stem cells are thus the key targets of 1) long-term and transient-regenerative/epigenetic gene therapy and 2) of recurrence-free anticancer therapy, respectively. While cancer stem cell gene therapy still needs time to accomplish, autologous stem cells have been instrumental in the first unequivocal successes for gene therapy whereby ex vivo retrovirally corrected hematopoietic stem cells have been returned to the patients.This timely book presents 1) the aforementioned stem cell gene therapy achievements that rely on random-integration of therapeutic transgenes into host chromosomes and 2) emerging experimental approaches aimed at eliminating random-integration oncogenic hazards through site-specific integration or gene targeting. Breakthrough endonuclease-boosted gene targeting for gene correction (inherited diseases) or targeted integration of therapeutic transgenes (other disorders) culminating in an efficiency compatible with clinical trials is one of the highlights of the book. Highlights also include the pioneering transplantation of adult pluripotent stem cells as a substitute for tissue-specific stem cells, thereby pinpointing the invaluable potential for stem cell gene therapy applications of autologous cells able to contribute to all three germ layers. Stem cell gene therapy is thus discussed in terms of 1) magnifying stem cell therapeutic homing through transient regenerative gene therapy and 2) of tackling most pathologies (including mitochondrial DNA diseases and ageing disorders) through stem cell repopulation dynamics into appropriate niches (long-term engraftment) and tissues (cell turn-over). Regarding cancer stem cell gene therapy, focus is on both the increasing number of identified tissue-specific cancer stem cells as the ultimate therapeutic targets and on the development of armed stem cells as tumor-homing vectors for targeted anticancer therapy.


Organoid Technology for Disease Modelling and Personalized Treatment

Organoid Technology for Disease Modelling and Personalized Treatment
Author: Badrul Hisham Yahaya
Publisher: Springer Nature
Total Pages: 209
Release: 2022-02-14
Genre: Science
ISBN: 3030930564

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Organoid Technology for Disease Modelling and Personalised Treatment provides a comprehensive overview of current knowledge of the organoid as a human-organ-in-a-dish, a powerful new technology for studying fundamental aspects of human organ development and disease progression in the search for drugs for personalised treatment. This preclinical tool is extensively being utilised as a model for studying human diseases in a dish, which is critical for accurate predictive modelling in precision medicine. The chapters in this book introduces readers to the numerous applications of organoids in various fields of study, as well as ethical considerations associated with organoids. In stem cell biology and regenerative medicine, where chimaera research, biomaterials for tissue vascularisation, gene-editing technologies, and their use in clinical procedures especially issues related to ethical concern over the use of human organoids have gotten much attention. Organoid Technology for Disease Modelling and Personalised Treatment is an excellent resource for in-depth research on one of the most interesting and significant topics in stem cell and regenerative medicine. This book's chapter collection covers a fresh viewpoint on organoid technology that scholars will require reading.


Human Genome Editing

Human Genome Editing
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 329
Release: 2017-08-13
Genre: Medical
ISBN: 0309452880

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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.