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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


Precision Medicine, CRISPR, and Genome Engineering

Precision Medicine, CRISPR, and Genome Engineering
Author: Stephen H. Tsang
Publisher: Springer
Total Pages: 178
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.


Autologous and Cancer Stem Cell Gene Therapy

Autologous and Cancer Stem Cell Gene Therapy
Author: Roger Bertolotti
Publisher: World Scientific
Total Pages: 489
Release: 2008
Genre: Medical
ISBN: 9812775862

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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.


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 Cell and Gene-Based Therapy

Stem Cell and Gene-Based Therapy
Author: Alexander Battler
Publisher: Springer Science & Business Media
Total Pages: 460
Release: 2007-06-26
Genre: Medical
ISBN: 9781846286766

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Regenerative medicine – stem cell and gene-based therapy – offers a new approach for restoring function of damaged organs and tissues. This is the first book to cover the major new aspects and field of regenerative medicine. This title is therefore a timely addition to the literature. It brings together the major approaches to regenerative medicine in one text, which ensures that techniques learnt in one discipline are disseminated across other areas of medicine.


Gene Therapy in Reconstructive and Regenerative Surgery

Gene Therapy in Reconstructive and Regenerative Surgery
Author: Giorgio Giatsidis
Publisher: Springer
Total Pages: 113
Release: 2018-08-02
Genre: Medical
ISBN: 3319789570

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This book offers an updated overview of the most recent research advances in the field, a comparison of established techniques and methods, a discussion on current experimental and translational challenges, and a commentary on potential opportunities and future directions. Dedicated chapters address and review the preclinical and clinical state-of-the-art of gene therapies for the reconstructive and regenerative surgery of skin and wounds, pathological scars, cartilage, tendons, skeletal muscles, and bio-engineered flaps. A brief guide to developing gene therapy clinical trials in the context of reconstructive and regenerative surgery is also provided. Biomedical and technological innovations are transforming our capacity to use gene therapies to safely and effectively repair, reconstruct, and regenerate tissues that are deficient or have been damaged by trauma and diseases. The targeted and controlled modulation of gene expression in tissues represents a game-changing, next-generation therapeutic tool for the modern reconstructive surgeon, expanding the horizon of regenerative surgery and tissue engineering. Through gene therapies, surgeons can direct (stem) cell differentiation and cell function, modulate the release of growth/transcriptional factors, affect the biological properties of regenerative scaffolds, control tissue inflammation, or induce immune-suppression in composite tissue allotransplants and xenotransplants. Written by renowned reconstructive surgeons and leading experts in each of these fields - from top academic institutions around the globe, the book provides an initial practical guide for veteran and newcomer surgeons alike, as well as for researchers interested in exploring the latest gene-based therapeutic strategies for reconstructive and regenerative surgery.


Trends in Stem Cell Biology and Technology

Trends in Stem Cell Biology and Technology
Author: Hossein Baharvand
Publisher: Springer Science & Business Media
Total Pages: 420
Release: 2009-04-20
Genre: Science
ISBN: 1603279059

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Stem cells, characterized by the ability to both self-renew and to generate diff- entiated functional cell types, have been derived from the embryo and from va- ous sources of the postnatal animals and human. The recent advances in stem cell research have led to a better understanding of self-renewal, maintenance, and diff- entiation of both embryonic and somatic stem cells. This has significantly increased our knowledge of cellular and developmental biology in general and will certainly continue to do so for a long time to come. Moreover, given their role in maintaining and replenishing tissues, stem cells represent a potential means of restoring tissue function and thereby treating the root cause of degenerative disease. Therefore, in parallel, we need to improve our cognizance of the challenges involved in applying stem cells in clinical settings. The current chapters highlight both of these aspects: that of understanding the “actual” and that of developing the “possible. ” In recognition of the growing excitement and potential of stem cells as models for both the advancement of basic science and future clinical applications, I felt it timely to edit this book in which forefront investigators would provide new findings for the use of stem cells to study various lineages and tissue types and some app- cations.


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.


Stem Cells in Regenerative Medicine

Stem Cells in Regenerative Medicine
Author: Julie Audet
Publisher: Methods in Molecular Biology
Total Pages: 472
Release: 2009
Genre: Science
ISBN:

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Here, leading experts in the field provide an updated representation of the landscape of stem cell-based therapies in a wide spectrum of tissue systems and ontogenic stages, from the isolation and culture of stem cells to their actual use in vivo.


Tissue Stem Cells

Tissue Stem Cells
Author: Andrew G. Renehan
Publisher: CRC Press
Total Pages: 424
Release: 2019-08-30
Genre:
ISBN: 9780367390921

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Tissue stem cells and their medical applications have become a major focus of research over the past decade. With 16 full-color illustrations, this reference provides a thorough and up-to-date overview of the current and emerging technologies for stem cell research and transplantation. Divided into three sections covering general issues, adult stem cells within specific tissues, and clinical applications, this source studies advances in bone marrow transplantation, cancer development modeling, tumor analysis, and gene therapy.