Heritable Human Genome Editing
Heritable Human Genome Editing

Author: The Royal Society

Publisher: National Academies Press

Published: 2021-01-16

Total Pages: 239

ISBN-13: 0309671132

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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.

Human Genome Editing
Human Genome Editing

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2017-08-13

Total Pages: 329

ISBN-13: 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.

Mutation-Specific Gene Editing for Blood Disorders
Mutation-Specific Gene Editing for Blood Disorders

Author: Carsten Werner Lederer

Publisher: Frontiers Media SA

Published: 2021-11-25

Total Pages: 218

ISBN-13: 2889716880

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Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com.

Genome Editing in Neurosciences
Genome Editing in Neurosciences

Author: Rudolf Jaenisch

Publisher:

Published: 2020-10-08

Total Pages: 128

ISBN-13: 9781013268564

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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.

CRISPR-Cas Systems
CRISPR-Cas Systems

Author: Rodolphe Barrangou

Publisher: Springer Science & Business Media

Published: 2012-12-13

Total Pages: 300

ISBN-13: 364234657X

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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.

Somatic Gene Therapy
Somatic Gene Therapy

Author: P.L. Chang

Publisher: CRC Press

Published: 2018-01-17

Total Pages: 242

ISBN-13: 1351093665

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As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.

Pharmaceutical Biotechnology
Pharmaceutical Biotechnology

Author: Daan J. A. Crommelin

Publisher: CRC Press

Published: 2002-11-14

Total Pages: 456

ISBN-13: 9780415285018

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The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.

Editing Humanity
Editing Humanity

Author: Kevin Davies

Publisher: Simon and Schuster

Published: 2020-10-06

Total Pages: 402

ISBN-13: 1643133942

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One of the world's leading experts on genetics unravels one of the most important breakthroughs in modern science and medicine. IIf our genes are, to a great extent, our destiny, then what would happen if mankind could engineer and alter the very essence of our DNA coding? Millions might be spared the devastating effects of hereditary disease or the challenges of disability, whether it was the pain of sickle-cell anemia to the ravages of Huntington’s disease. But this power to “play God” also raises major ethical questions and poses threats for potential misuse. For decades, these questions have lived exclusively in the realm of science fiction, but as Kevin Davies powerfully reveals in his new book, this is all about to change. Engrossing and page-turning, Editing Humanity takes readers inside the fascinating world of a new gene editing technology called CRISPR, a high-powered genetic toolkit that enables scientists to not only engineer but to edit the DNA of any organism down to the individual building blocks of the genetic code. Davies introduces readers to arguably the most profound scientific breakthrough of our time. He tracks the scientists on the front lines of its research to the patients whose powerful stories bring the narrative movingly to human scale. Though the birth of the “CRISPR babies” in China made international news, there is much more to the story of CRISPR than headlines seemingly ripped from science fiction. In Editing Humanity, Davies sheds light on the implications that this new technology can have on our everyday lives and in the lives of generations to come.

Gene Editing
Gene Editing

Author: Yuan-Chuan Chen

Publisher: BoD – Books on Demand

Published: 2019-05-29

Total Pages: 110

ISBN-13: 1789845084

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Gene-editing technologies (e.g., ZFNs, TALENs, and CRISPRs/Cas9) have been extensively used as tools in basic research. They are further applied in manufacturing agricultural products, food, industrial products, medicinal products, etc. Particularly, the discovery of medicinal products using gene-editing technologies will open a new era for human therapeutics. Though there are still many technical and ethical challenges ahead of us, more and more products based on gene-editing technologies have been approved for marketing. These technologies are promising for multiple applications. Their development and implications should be explored in the broadest context possible. Future research directions should also be highlighted. In this book, the applications, perspectives, and challenges of gene-editing technologies are significantly demonstrated and discussed.