Gene Therapy For Acute And Acquired Diseases

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Immunopharmacology

Author : Manzoor M. Khan
Publisher : Springer Science & Business Media
ISBN 13 : 0387779760
Total Pages : 275 pages
Book Rating : 4.3/5 (877 download)

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Book Synopsis Immunopharmacology by : Manzoor M. Khan

Download or read book Immunopharmacology written by Manzoor M. Khan and published by Springer Science & Business Media. This book was released on 2008-12-19 with total page 275 pages. Available in PDF, EPUB and Kindle. Book excerpt: During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

Gene Therapy for Acute and Acquired Diseases

Author : Phillip H. Factor
Publisher : Springer Science & Business Media
ISBN 13 : 1461516676
Total Pages : 312 pages
Book Rating : 4.4/5 (615 download)

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Book Synopsis Gene Therapy for Acute and Acquired Diseases by : Phillip H. Factor

Download or read book Gene Therapy for Acute and Acquired Diseases written by Phillip H. Factor and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 312 pages. Available in PDF, EPUB and Kindle. Book excerpt: In recent years much enthusiasm and energy has been directed toward the development of human gene therapies, especially for inherited conditions and cancers. However, current gene transfer technology is limited in its transduction efficiency and ability to permanently and safely correct genomic defects. Thus the promise of gene therapy for these conditions is as yet unrealized. The progression of gene transfer technology will eventually surmount these limitations. Gene Therapy for Acute and Acquired Diseases includes selected examples of ongoing studies in molecular genetics that have the potential to evolve into human therapies for acute illnesses. These chapters are intended to highlight lesser known applications of gene therapy for acquired disorders. It is expected that human gene therapy trials for these conditions will be forthcoming in the near future, leading to previously unimaginable therapies. Thus, this first-ever book about gene therapy for acute and acquired diseases is intended to serve as a glimpse into the future.

Polymers and Nanomaterials for Gene Therapy

Author : Ravin Narain
Publisher : Woodhead Publishing
ISBN 13 : 0081005210
Total Pages : 303 pages
Book Rating : 4.0/5 (81 download)

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Book Synopsis Polymers and Nanomaterials for Gene Therapy by : Ravin Narain

Download or read book Polymers and Nanomaterials for Gene Therapy written by Ravin Narain and published by Woodhead Publishing. This book was released on 2016-01-09 with total page 303 pages. Available in PDF, EPUB and Kindle. Book excerpt: Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. - Explores current challenges in the research of genetic diseases - Discusses polymers for gene therapy and their function in designing advanced materials - Provides examples of organic and inorganic nanomaterials for gene therapy - Includes labeling, targeting, and assays - Looks at characterization, physico-(bio)chemical properties, and applications

Gene Therapy for Viral Infections

Author : Patrick Arbuthnot
Publisher : Academic Press
ISBN 13 : 0124114520
Total Pages : 391 pages
Book Rating : 4.1/5 (241 download)

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Book Synopsis Gene Therapy for Viral Infections by : Patrick Arbuthnot

Download or read book Gene Therapy for Viral Infections written by Patrick Arbuthnot and published by Academic Press. This book was released on 2015-06-01 with total page 391 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Therapy for Viral Infections provides a comprehensive review of the broader field of nucleic acid and its use in treating viral infections. The text bridges the gap between basic science and important clinical applications of the technology, providing a systematic, integrated review of the advances in nucleic acid-based antiviral drugs and the potential advantages of new technologies over current treatment options. Coverage begins with the fundamentals, exploring varying topics, including harnessing RNAi to silence viral gene expression, antiviral gene editing, viral gene therapy vectors, and non-viral vectors. Subsequent sections include detailed coverage of the developing use of gene therapy for the treatment of specific infections, the principles of rational design of antivirals, and the hurdles that currently face the further advancement of gene therapy technology. - Provides coverage of gene therapy for a variety of infections, including HBV, HCV, HIV, hemorrhagic fever viruses, and respiratory and other viral infections - Bridges the gap between the basic science and the important medical applications of this technology - Features a broad approach to the topic, including an essential overview and the applications of gene therapy, synthetic RNA, and other antiviral strategies that involve nucleic acid engineering - Presents perspectives on the future use of nucleic acids as a novel class of antiviral drugs - Arms the reader with the cutting-edge information needed to stay abreast of this developing field

Rare Diseases and Orphan Products

Author : Institute of Medicine
Publisher : National Academies Press
ISBN 13 : 0309158060
Total Pages : 442 pages
Book Rating : 4.3/5 (91 download)

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Book Synopsis Rare Diseases and Orphan Products by : Institute of Medicine

Download or read book Rare Diseases and Orphan Products written by Institute of Medicine and published by National Academies Press. This book was released on 2011-04-03 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

CRISPR-Cas Systems

Author : Rodolphe Barrangou
Publisher : Springer Science & Business Media
ISBN 13 : 364234657X
Total Pages : 300 pages
Book Rating : 4.6/5 (423 download)

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Book Synopsis CRISPR-Cas Systems by : Rodolphe Barrangou

Download or read book CRISPR-Cas Systems written by Rodolphe Barrangou and published by Springer Science & Business Media. This book was released on 2012-12-13 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Gene Therapy of Autoimmune Disease

Author : Gerald J. Prud'homme
Publisher : Springer
ISBN 13 : 9780306479915
Total Pages : 149 pages
Book Rating : 4.4/5 (799 download)

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Book Synopsis Gene Therapy of Autoimmune Disease by : Gerald J. Prud'homme

Download or read book Gene Therapy of Autoimmune Disease written by Gerald J. Prud'homme and published by Springer. This book was released on 2005-07-13 with total page 149 pages. Available in PDF, EPUB and Kindle. Book excerpt: Autoimmune diseases are diverse and responsible for considerable morbidity. Their etiology remains largely unknown, and current therapy with anti-inflammatory drugs is prone to adverse effects, and rarely curative. New therapies with anti-cytokine antibodies or receptors are promising, but require frequent administration of expensive protein drugs. Gene Therapy of Autoimmune Diseases comprehensively reviews research in gene therapy for autoimmune diseases with viral or non-viral vectors. Gene therapy offers the possibility of long-term, continuous delivery of a wide variety of immunosuppressive, anti-inflammatory, or tolerance-inducing agents. Moreover, highly specific genetically modified cells can be produced. This book discusses the most promising avenues in this exciting new field.

Human Genome Editing

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309452880
Total Pages : 329 pages
Book Rating : 4.3/5 (94 download)

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Book Synopsis Human Genome Editing by : National Academies of Sciences, Engineering, and Medicine

Download or read book Human Genome Editing written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2017-08-13 with total page 329 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Acute Respiratory Distress Syndrome

Author : Sadis Matalon
Publisher : Springer Science & Business Media
ISBN 13 : 1441986340
Total Pages : 428 pages
Book Rating : 4.4/5 (419 download)

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Book Synopsis Acute Respiratory Distress Syndrome by : Sadis Matalon

Download or read book Acute Respiratory Distress Syndrome written by Sadis Matalon and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 428 pages. Available in PDF, EPUB and Kindle. Book excerpt: Proceedings of a NATO ASI held in Corfu, Greece, June 15-25, 1997

Gene Therapy for Diseases of the Lung

Author : Kenneth Brigham
Publisher : CRC Press
ISBN 13 : 9780824700607
Total Pages : 460 pages
Book Rating : 4.7/5 (6 download)

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Book Synopsis Gene Therapy for Diseases of the Lung by : Kenneth Brigham

Download or read book Gene Therapy for Diseases of the Lung written by Kenneth Brigham and published by CRC Press. This book was released on 1997-04-10 with total page 460 pages. Available in PDF, EPUB and Kindle. Book excerpt: This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems.

Childhood Acute Lymphoblastic Leukemia

Author : Ajay Vora
Publisher : Springer
ISBN 13 : 3319397087
Total Pages : 345 pages
Book Rating : 4.3/5 (193 download)

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Book Synopsis Childhood Acute Lymphoblastic Leukemia by : Ajay Vora

Download or read book Childhood Acute Lymphoblastic Leukemia written by Ajay Vora and published by Springer. This book was released on 2017-04-21 with total page 345 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a comprehensive and up-to-date review of all aspects of childhood Acute Lymphoblastic Leukemia, from basic biology to supportive care. It offers new insights into the genetic pre-disposition to the condition and discusses how response to early therapy and its basic biology are utilized to develop new prognostic stratification systems and target therapy. Readers will learn about current treatment and outcomes, such as immunotherapy and targeted therapy approaches. Supportive care and management of the condition in resource poor countries are also discussed in detail. This is an indispensable guide for research and laboratory scientists, pediatric hematologists as well as specialist nurses involved in the care of childhood leukemia.

Pharmaceutical Biotechnology

Author : Daan J. A. Crommelin
Publisher : CRC Press
ISBN 13 : 9780415285018
Total Pages : 456 pages
Book Rating : 4.2/5 (85 download)

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Book Synopsis Pharmaceutical Biotechnology by : Daan J. A. Crommelin

Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Gene Therapy for Diseases of the Lung

Author : Kenneth Brigham
Publisher : CRC Press
ISBN 13 : 1000145921
Total Pages : 460 pages
Book Rating : 4.0/5 (1 download)

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Book Synopsis Gene Therapy for Diseases of the Lung by : Kenneth Brigham

Download or read book Gene Therapy for Diseases of the Lung written by Kenneth Brigham and published by CRC Press. This book was released on 2020-10-14 with total page 460 pages. Available in PDF, EPUB and Kindle. Book excerpt: This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems.

Genetics of Bone Biology and Skeletal Disease

Author : Rajesh V. Thakker
Publisher : Academic Press
ISBN 13 : 0128041986
Total Pages : 880 pages
Book Rating : 4.1/5 (28 download)

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Book Synopsis Genetics of Bone Biology and Skeletal Disease by : Rajesh V. Thakker

Download or read book Genetics of Bone Biology and Skeletal Disease written by Rajesh V. Thakker and published by Academic Press. This book was released on 2017-10-31 with total page 880 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genetics of Bone Biology and Skeletal Disease, Second Edition, is aimed at students of bone biology and genetics and includes general introductory chapters on bone biology and genetics. More specific disease orientated chapters comprehensively summarize the clinical, genetic, molecular, animal model, molecular pathology, diagnostic, counseling, and treatment aspects of each disorder. The book is organized into five sections that each emphasize a particular theme, general background to bone biology, general background to genetics and epigenetics, disorders of bone and joint, parathyroid and related disorders, and vitamin D and renal disorders. The first section is specifically devoted to providing an overview of bone biology and structure, joint and cartilage biology, principles of endocrine regulation of bone, and the role of neuronal regulation and energy homeostasis. The second section reviews the principles and progress of medical genetics and epigenetics related to bone disease, including genome-wide association studies (GWAS), genomic profiling, copy number variation, prospects of gene therapy, pharmacogenomics, genetic testing and counseling, as well as the generation and utilizing of mouse models. The third section details advances in the genetics and molecular biology of bone and joint diseases, both monogenic and polygenic, as well as skeletal dysplasias, and rarer bone disorders. The fourth section highlights the central role of the parathyroids in calcium and skeletal homeostasis by reviewing the molecular genetics of: hyperparathyroidism, hypoparathyrodism, endocrine neoplasias, and disorders of the PTH and calcium-sensing receptors. The fifth section details molecular and cellular advances across associated renal disorders such as vitamin D and rickets. - Identifies and analyzes the genetic basis of bone disorders in humans and demonstrates the utility of mouse models in furthering the knowledge of mechanisms and evaluation of treatments - Demonstrates how the interactions between bone and joint biology, physiology, and genetics have greatly enhanced the understanding of normal bone function as well as the molecular pathogenesis of metabolic bone disorders - Summarizes the clinical, genetic, molecular, animal model, molecular pathology, diagnostic, counseling, and treatment aspects of each disorder

A Guide to Human Gene Therapy

Author : Roland W. Herzog
Publisher : World Scientific
ISBN 13 : 9814280917
Total Pages : 415 pages
Book Rating : 4.8/5 (142 download)

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Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog

Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne

Encyclopedia of Genetics, Genomics, Proteomics, and Informatics

Author : George P. Rédei
Publisher : Springer Science & Business Media
ISBN 13 : 1402067534
Total Pages : 2139 pages
Book Rating : 4.4/5 (2 download)

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Book Synopsis Encyclopedia of Genetics, Genomics, Proteomics, and Informatics by : George P. Rédei

Download or read book Encyclopedia of Genetics, Genomics, Proteomics, and Informatics written by George P. Rédei and published by Springer Science & Business Media. This book was released on 2008-04-25 with total page 2139 pages. Available in PDF, EPUB and Kindle. Book excerpt: This new third edition updates a best-selling encyclopedia. It includes about 56% more words than the 1,392-page second edition of 2003. The number of illustrations increased to almost 2,000 and their quality has improved by design and four colors. It includes approximately 1,800 current databases and web servers. This encyclopedia covers the basics and the latest in genomics, proteomics, genetic engineering, small RNAs, transcription factories, chromosome territories, stem cells, genetic networks, epigenetics, prions, hereditary diseases, and patents. Similar integrated information is not available in textbooks or on the Internet.

The Gene

Author : Siddhartha Mukherjee
Publisher : Simon and Schuster
ISBN 13 : 1476733538
Total Pages : 624 pages
Book Rating : 4.4/5 (767 download)

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Book Synopsis The Gene by : Siddhartha Mukherjee

Download or read book The Gene written by Siddhartha Mukherjee and published by Simon and Schuster. This book was released on 2016-05-17 with total page 624 pages. Available in PDF, EPUB and Kindle. Book excerpt: The #1 NEW YORK TIMES Bestseller The basis for the PBS Ken Burns Documentary The Gene: An Intimate History Now includes an excerpt from Siddhartha Mukherjee’s new book Song of the Cell! From the Pulitzer Prize–winning author of The Emperor of All Maladies—a fascinating history of the gene and “a magisterial account of how human minds have laboriously, ingeniously picked apart what makes us tick” (Elle). “Sid Mukherjee has the uncanny ability to bring together science, history, and the future in a way that is understandable and riveting, guiding us through both time and the mystery of life itself.” —Ken Burns “Dr. Siddhartha Mukherjee dazzled readers with his Pulitzer Prize-winning The Emperor of All Maladies in 2010. That achievement was evidently just a warm-up for his virtuoso performance in The Gene: An Intimate History, in which he braids science, history, and memoir into an epic with all the range and biblical thunder of Paradise Lost” (The New York Times). In this biography Mukherjee brings to life the quest to understand human heredity and its surprising influence on our lives, personalities, identities, fates, and choices. “Mukherjee expresses abstract intellectual ideas through emotional stories…[and] swaddles his medical rigor with rhapsodic tenderness, surprising vulnerability, and occasional flashes of pure poetry” (The Washington Post). Throughout, the story of Mukherjee’s own family—with its tragic and bewildering history of mental illness—reminds us of the questions that hang over our ability to translate the science of genetics from the laboratory to the real world. In riveting and dramatic prose, he describes the centuries of research and experimentation—from Aristotle and Pythagoras to Mendel and Darwin, from Boveri and Morgan to Crick, Watson and Franklin, all the way through the revolutionary twenty-first century innovators who mapped the human genome. “A fascinating and often sobering history of how humans came to understand the roles of genes in making us who we are—and what our manipulation of those genes might mean for our future” (Milwaukee Journal-Sentinel), The Gene is the revelatory and magisterial history of a scientific idea coming to life, the most crucial science of our time, intimately explained by a master. “The Gene is a book we all should read” (USA TODAY).