Download or read book Preclinical Studies Towards Stem Cell Gene Therapy for Gaucher Diseasse written by Menzo Jans Emco Havenga and published by . This book was released on 1999 with total page 172 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book The Neuronal Ceroid Lipofuscinoses Batten Disease written by Sara Mole and published by OUP Oxford. This book was released on 2011-03-10 with total page 772 pages. Available in PDF, EPUB and Kindle. Book excerpt: The neuronal ceroid lipofuscinoses are an extremely rare group of inherited neurodegenerative diseases that primarily affect children. Core symptoms of these conditions typically include epilepsy, cognitive decline and visual failure. These diseases are so rare that professionals who come into contact with them need a consultative reference work that enables them to become expert, or identify who to contact for more details. Fully updated and revised, this second edition continues to be the definitive volume on this devastating group of disorders. Written by an international collection of authorities in the field, it provides invaluable advice on their diagnosis, patient care, and new treatments that are available. This new edition of the definitive reference text on the neuronal ceroid lipofuscinoses will prove useful for clinicians, family physicians, research scientists, diagnostic laboratories, families affected by the disease as well as by workers in industry planning translational research.
Download or read book Stem Cell Biology and Gene Therapy written by Peter J. Quesenberry and published by John Wiley & Sons. This book was released on 1998-09-10 with total page 586 pages. Available in PDF, EPUB and Kindle. Book excerpt: STEM CELL BIOLOGY AND GENE THERAPY Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide. Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies. This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including: * Fundamental regulatory mechanisms that operate in stem cells * Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy * Delivery systems for therapeutic genes, including an overview of the most promising vectors * Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine.
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 Heritable Human Genome Editing written by The Royal Society and published by National Academies Press. This book was released on 2021-01-16 with total page 239 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book A Special Report on Gene Therapy written by K. K. Jain and published by John Wiley & Sons. This book was released on 2000-04-14 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt: This special report on Gene Therapy Companies by K.K. Jain provides an up-to-date overview of the gene therapy industry. It contains a wealth of very practical information on 105 companies. For each company, information is available on the people to contact, their contact details, a corporate history, the company's technologies and products, financial data, patents held, products in clinical trials, collaborations with other companies and selected publications from the company's scientists. Almost 200 companies were initially considered for inclusion but just over 100 companies were finally selected as they fulfilled the necessary criteria to be classified as a 'Gene Therapy Company,', defined broadly to include cell therapy companies. Not all of the companies devote their activity exclusively to gene therapy but they have sufficient relevance to be included. As part of this project, new companies were discovered that have not been reported in other publications or on the internet. Dr. Jain has spared no effort to ensure that the information is up-to-date and accurate at the time of publishing. In most instances, the information was reviewed by the companies to correct any outdated information or to add any complementary or recent information. In addition to the detailed descriptions of each company, the data is also presented in summary tabular form so that at a glance the reader can identify collaborations within the gene therapy industry, or determine which companies are pursuing strategies involving viral, non-viral or other approaches to gene therapy, and which diseases are being targetted. Given that much of the gene therapy research and development is currently being carried out within the industrial sector, it is important for scientists, managers and investors to have the relevant information at their fingertips. This special report provides that information in one accurate, easy to consult and up-to-date source.
Download or read book Gene Therapy of Cancer written by Stanton L. Gerson and published by Elsevier. This book was released on 2002-04-04 with total page 555 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. - Provides in-depth description of targeted systems and treatment strategies - Explains the underlying cancer biology necessary for understanding a given therapeutic approach - Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses - Presents translational focus with emphasis on requirements for clinical implementation - Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference
Download or read book Hematopoietic Stem Cell Therapy written by Edward David Ball and published by . This book was released on 2000 with total page 794 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book will be the only current practical guide to a widely used procedure for treating leukemias and disseminated cancers. The contents are organized chronologically, to serve as a step-by-step guide throughout the transplant process. Comprehensive yet concise, it emphasizes the latest techniques, such as peripheral blood stem cell grafts.
Download or read book Hematopoietic Stem Cell Transplantation written by Anthony D. Ho and published by CRC Press. This book was released on 2000-07-25 with total page 610 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book integrates recent advances in molecular and cell biology of hematopoietic stem cells (HSC) with developments in clinical research in stem cell-based therapy-providing an up-to-date review of novel cytokines and cellular components; animal models; cell preparation, selection, and collection; minimal residual disease and purging; expansion
Download or read book Wintrobe s Clinical Hematology written by John P. Greer and published by Lippincott Williams & Wilkins. This book was released on 2013-08-29 with total page 3094 pages. Available in PDF, EPUB and Kindle. Book excerpt: With the 13th edition, Wintrobe’s Clinical Hematology once again bridges the gap between the clinical practice of hematology and the basic foundations of science. Broken down into eight parts, this book provides readers with a comprehensive overview of: Laboratory Hematology, The Normal Hematologic System, Transfusion Medicine, Disorders of Red Cells, Hemostasis and Coagulation; Benign Disorders of Leukocytes, The Spleen and/or Immunoglobulins; Hematologic Malignancies, and Transplantation. Within these sections, there is a heavy focus on the morphological exam of the peripheral blood smear, bone marrow, lymph nodes, and other tissues. With the knowledge about gene therapy and immunotherapy expanding, new, up-to-date information about the process and application of these therapies is included. Likewise, the editors have completely revised material on stem cell transplantation in regards to both malignant and benign disorders, graft versus host disease, and the importance of long-term follow-up of transplantation survivors.
Download or read book Recombinant DNA Research written by and published by . This book was released on 1995 with total page 940 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Gaucher Disease written by Anthony H. Futerman and published by CRC Press. This book was released on 2006-07-07 with total page 553 pages. Available in PDF, EPUB and Kindle. Book excerpt: In September of 2007 Gaucher Disease received a commendation in the Haematology category of the 2007 British Medical Association Medical Book Competition! Although rare in the general population, Gaucher disease is the most prevalent of the lysosomal storage disorders, making research into this particular orphan disorder an invaluable proto
Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.
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 Cell and Gene Therapies for Neurologic Diseases written by and published by Elsevier. This book was released on 2024-10-02 with total page 342 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cell and Gene Therapies for Neurologic Diseases, Volume 205 comprehensively covers the scientific background, translational efforts, clinical developments and registered biologics that have entered into clinical practice. Coverage includes types of therapies available and in development, and best practice uses for a variety of neurological disorders including Parkinson's, Huntington's, ALS, stroke, spinal cord RP, demyelination, and epilepsy. As the emergence of gene and cellular therapeutics has changed the clinical landscape for a variety of disorders, and is now ready to do so for neurological diseases, these therapeutic modalities currently complement, and may in time, supplant small molecule drugs. - Summarizes advances in cell and gene therapy for neurological diseases - Describes the therapies available and in development - Includes surgical, ethical, and manufacturing considerations - Identifies best practices for specific neurological diseases - Covers Huntington's, Parkinson's, ALS, Stroke, Demyelination, epilepsy, and more
Download or read book Thomas Hematopoietic Cell Transplantation written by Stephen J. Forman and published by John Wiley & Sons. This book was released on 2015-12-14 with total page 1416 pages. Available in PDF, EPUB and Kindle. Book excerpt: Fully revised for the fifth edition, this outstanding reference on bone marrow transplantation is an essential, field-leading resource. Extensive coverage of the field, from the scientific basis for stem-cell transplantation to the future direction of research Combines the knowledge and expertise of over 170 international specialists across 106 chapters Includes new chapters addressing basic science experiments in stem-cell biology, immunology, and tolerance Contains expanded content on the benefits and challenges of transplantation, and analysis of the impact of new therapies to help clinical decision-making Includes a fully searchable Wiley Digital Edition with downloadable figures, linked references, and more References for this new edition are online only, accessible via the Wiley Digital Edition code printed inside the front cover or at www.wiley.com/go/forman/hematopoietic.
Download or read book Muscle Biopsy written by Victor Dubowitz and published by Bailliere Tindall Limited. This book was released on 1985 with total page 746 pages. Available in PDF, EPUB and Kindle. Book excerpt:
