Download or read book The Thalassemias written by D. J. Weatherall and published by . This book was released on 1983 with total page 186 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Lentiviral Vectors written by Didier Trono and published by Springer Science & Business Media. This book was released on 2001-12-14 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: For the first time a compilation of chapters that depict the biological bases underlying the development of lentiviral vectors, the techniques involved in the manufacture of this new gene delivery tool, and its most promising applications.

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

Download or read book Gene Therapy Protocols written by Joseph LeDoux and published by Humana Press. This book was released on 2008-04-24 with total page 408 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors and Design and Characterization of Gene Transfer Vectors. Readers will find a comprehensive resource of current and emerging methods for the production of viral and non-viral gene transfer vectors, as well as detailed protocols for applications in stem cell biology, cancer research and infectious disease.

Download or read book Thalassemia and Other Hemolytic Anemias written by Isam Jaber Al-Zwaini and published by BoD – Books on Demand. This book was released on 2018-07-11 with total page 140 pages. Available in PDF, EPUB and Kindle. Book excerpt: Thalassemia is a very common disease first described by pediatrician Thomas Benton Cooley in 1925 who described it in a patient of Italian origin. At that time, it was designated as Cooley's anemia. George Hoyt Whipple, a Nobel prize winner, and W. L. Bradford, a professor of pediatrics at the University of Rochester, coined the term thalassemia in 1936, which in Greek means anemia of the sea (Thalassa means "sea", and emia means "blood"), due to the fact that it is very common in the area of the Mediterranean Sea. This name is actually misleading because it can occur everywhere in the world. Thalassemia is not a single disease; it is rather a group of hereditary disorders of the production of globulin chain of the hemoglobin. Throughout the world, thalassemia affects approximately 4.4 of every 10,000 live births. It represents a major social and emotional impact on the patient and his family and a major burden on health services where the prevalence is high.

Download or read book Advanced Textbook On Gene Transfer Gene Therapy And Genetic Pharmacology Principles Delivery And Pharmacological And Biomedical Applications Of Nucleotide based Therapies Second Edition written by Daniel Scherman and published by World Scientific. This book was released on 2019-07-16 with total page 634 pages. Available in PDF, EPUB and Kindle. Book excerpt: This unique advanced textbook provides a clear and comprehensive overview of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations. The second edition features new groundbreaking material on genome editing using the recently discovered CRISPR/Cas9 system and on cancer immunotherapy by CAR-T cells. It also presents the historical milestone of gene therapy application in the field of severe combined immunodeficiency, and other fields of gene therapy and molecular medicine.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of gene therapy, which is already revolutionizing medicine. In this advanced textbook, more than 30 leading scientists come together to explore these topics.This educational introduction provides the background material needed to further explore the subject as well as relevant research literature. It is an invaluable resource to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide or develop a gene therapy strategy. The second edition's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome.

Download or read book Cell and Gene Therapies written by Miguel-Angel Perales and published by Springer. This book was released on 2018-11-27 with total page 286 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Download or read book Sickle Cell Disease and Hematopoietic Stem Cell Transplantation written by Emily Riehm Meier and published by Springer. This book was released on 2017-09-19 with total page 337 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a comprehensive, state-of-the art review of hematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD). The book reviews new data about risk prediction for severe SCD, outlines the unique challenges of HSCT for patients with SCD, profiles the supportive care guidelines for patients who are undergoing HSCT, highlights our current understanding of the best transfusion support for SCD patients prior to, during and after HSCT, and provides new perspectives about the ethics of HSCT for pediatric patients with SCD. Published in the last few years, several landmark phase III trials that utilize matched unrelated and haploidentical donors for HSCT in SCD patients are also placed in context with respect to current management. Written by experts in the field, Sickle Cell Disease and Hematopoietic Stem Cell Transplantation is a valuable resource for physicians and researchers dealing with and interested in this challenging, yet exciting, curative therapy for sickle cell disease, that will help guide patient management and stimulate investigative efforts.

Download or read book Vogel and Motulsky s Human Genetics written by Michael Speicher and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 1006 pages. Available in PDF, EPUB and Kindle. Book excerpt: The fourth edition of this classical reference book can once again be relied upon to present a cohesive and up-to-date exposition of all aspects of human and medical genetics. Human genetics has become one of the main basic sciences in medicine, and molecular genetics is increasingly becoming a major part of this field. This new edition integrates a wealth of new information - mainly describing the influence of the "molecular revolution" - including the principles of epigenetic processes which together create the phenotype of a human being. Other revisions are an improved layout, sub-division into a larger number of chapters, as well as two-colour print throughout for ease of reference, and many of the figures are now in full colour. For graduates and those already working in medical genetics.

Download or read book Gene and Cell Therapies for Beta Globinopathies written by Punam Malik and published by Springer. This book was released on 2017-11-09 with total page 254 pages. Available in PDF, EPUB and Kindle. Book excerpt: Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, which are often available only to 15-20% of patients. An alternative to allogeneic HS CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances.

Download or read book Gene Therapy Methods written by M. Ian Phillips and published by Academic Press. This book was released on 2002-02-22 with total page 770 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease

Download or read book The Business of Healthcare Innovation written by Lawton Robert Burns and published by Cambridge University Press. This book was released on 2005-08-25 with total page 408 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Business of Healthcare Innovation is the first wide-ranging analysis of business trends in the manufacturing segment of the health care industry. In this leading edge volume, Professor Burns focuses on the key role of the 'producers' as the main source of innovation in health systems. Written by professors of the Wharton School and industry executives, this book provides a detailed overview of the pharmaceutical, biotechnology, genomics/proteomics, medical device and information technology sectors. It analyses the market structures of these sectors as well as the business models and corporate strategies of firms operating within them. Most importantly, the book describes the growing convergence between these sectors and the need for executives in one sector to increasingly draw upon trends in the others. It will be essential reading for students and researchers in the field of health management, and of great interest to strategy scholars, industry practitioners and management consultants.

Download or read book The Cell Biology of Stem Cells written by Eran Meshorer and published by Springer Science & Business Media. This book was released on 2011-01-11 with total page 246 pages. Available in PDF, EPUB and Kindle. Book excerpt: Stem cells have been gaining a lot of attention in recent years. Their unique potential to self-renew and differentiate has turned them into an attractive model for the study of basic biological questions such as cell division, replication, transcription, cell fate decisions, and more. With embryonic stem (ES) cells that can generate each cell type in the mammalian body and adult stem cells that are able to give rise to the cells within a given lineage, basic questions at different developmental stages can be addressed. Importantly, both adult and embryonic stem cells provide an excellent tool for cell therapy, making stem cell research ever more pertinent to regenerative medicine. As the title The Cell Biology of Stem Cells suggests, our book deals with multiple aspects of stem cell biology, ranging from their basic molecular characteristics to the in vivo stem cell trafficking of adult stem cells and the adult stem-cell niche, and ends with a visit to regeneration and cell fate reprogramming. In the first chapter, “Early embryonic cell fate decisions in the mouse”, Amy Ralson and Yojiro Yamanaka describe the mechanisms that support early developmental decisions in the mouse pre-implantation embryo and the current understanding of the source of the most immature stem cell types, which includes ES cells, trophoblast stem (TS) cells and extraembryonic endoderm stem (XEN) cells.

Download or read book Essentials of Medical Genetics for Health Professionals written by Laura M. Gunder McClary and published by Jones & Bartlett Publishers. This book was released on 2010-09-17 with total page 249 pages. Available in PDF, EPUB and Kindle. Book excerpt: Essentials of Medical Genetics for Health Professionals is a concise, accessible introduction to medical genetics for all health professions students. Even with limited exposure to genetics, students can use the accelerated approach in this text to attain a base foundation of genetics knowledge. This book begins with a review of chromosomes, DNA, RNA, protein synthesis, and inheritance patterns and continues with a clinical focus based on understanding different disease processes. A variety of genetic diseases are explored, including what is known about the genetics involved, the signs and symptoms of the disease, and the treatment options available. Accompanying tables and images aid comprehension. This book also covers diagnostic techniques and an overview of embryonic development and teratogens. The roles of genetic counseling and screening, as well as the ethical and legal issues related to genetic screening and genetic testing are also discussed. Complete with stated objectives, definition of key terms, references, chapter summaries and end of chapter review questions with answers, each chapter is organized for optimal learning. Essentials of Medical Genetics for Health Professionals will not only have application in the classroom setting for health professions or medical students, but practicing clinicians such as physician assistants, nurse practitioners, and physicians who want to learn more or revisit genetics will also find this book a valuable, useful resource. Instructor Resources include PowerPoint Slides, a TestBank, and an Image Bank.

Download or read book An Introduction to Molecular Medicine and Gene Therapy written by Thomas F. Kresina and published by John Wiley & Sons. This book was released on 2004-03-24 with total page 408 pages. Available in PDF, EPUB and Kindle. Book excerpt: An Introduction to Molecular Medicine and Gene Therapy Edited by Thomas F. Kresina, Ph.D. Gene therapy, or the use of genetic manipulation for disease treatment, is derived from advances in genetics, molecular biology, clinical medicine, and human genomics. Molecular medicine, the application of molecular biological techniques to disease treatment and diagnosis, is derived from the development of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular Medicine and Gene Therapy provides a basis for interpreting new clinical and basic research findings in the areas of cloning, gene transfer, and targeting; the applications of genetic medicine to clinical conditions; ethics and governmental regulations; and the burgeoning fields of genomics, biotechnology, and bioinformatics. By dividing the material into three sections - an introduction to basic science, a review of clinical applications, and a discussion of the evolving issues related to gene therapy and molecular medicine-this comprehensive manual describes the basic approaches to the broad range of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine and Gene Therapy: * Covers new frontiers in gene therapy, animal models, vectors, gene targeting, and ethical/legal considerations * Provides organ-based reviews of current studies in gene therapy for monogenetic, multifactoral or polygenic disorders, and infectious diseases * Includes bold-faced terms, key concepts, summaries, and lists of helpful references by subject in each chapter * Contains appendices on commercial implications and a review of the history of gene therapy This textbook offers a clear, concise writing style, drawing upon the expertise of the authors, all renowned researchers in their respective specialties of molecular medicine. Researchers in genetics and molecular medicine will all find An Introduction to Molecular Medicine and Gene Therapy to be an essential guide to the rapidly evolving field of gene therapy and its applications in molecular medicine.

Download or read book Disorders of Hemoglobin written by Martin H. Steinberg and published by Cambridge University Press. This book was released on 2009-08-17 with total page 883 pages. Available in PDF, EPUB and Kindle. Book excerpt: Completely revised new edition of the definitive reference on disorders of hemoglobin.

Download or read book Prenatal Gene Therapy written by Charles Coutelle and published by Humana Press. This book was released on 2012-05-31 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions. In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly successful Methods in Molecular BiologyTM series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls. Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine.