Download or read book Retroviral Mediated Gene Transfer Into Hematopoietic Stem Cells as a Technique to Combat Caucher Disease written by Erin Ewing and published by . This book was released on 1994 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Genetic Modification of Hematopoietic Stem Cells written by Christopher Baum and published by Humana Press. This book was released on 2011-01-20 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Transfer into Hematopoietic Cells: From Basic Science to Clinical Application Christopher Baum 1. The Potential of Gene Transfer into Hematopoietic Vectors with the potential for stable transgene integration are Cells widely used in basic hematology and clinical trials of gene me- cine. In basic research, both gain-of-function and loss-of-fu- tion situations of individual genes can be created by gene transfer, leading to a wide range of applications in developmental biology, stem cell biology, immunology, leukemia research, and human genetics. With the first evidence of successful modification of murine hematopoietic cells using retroviral gene vectors (1, 2), researchers have also explored the therapeutic potential of this approach. To date, the emerging discipline of gene therapy is a highly diversified field that offers entirely novel approaches to treat a great variety of human diseases (3). All hematopoietic cell types are of major interest in this context, since the modification of the hematopoietic stem cell population may potentially give rise to a completely transgenic hematopoiesis with the potential to cure genetic disorders or fight severe chronic infections, and the targeting of mature cells such as lymphocytes or antigen-p- senting dendritic cells offers all types of transient and semiper- nent modifications of the immune system. The unifying principle of gene medicine is the need to transfer complex nucleic acids cells that do not contribute to the germline (somatic cells).

Download or read book In Vivo Expression of Human Glucocerebrosidase Following Retroviral mediated Gene Transfer Into Murine Hematopoietic Stem Cells written by Pamela Hankey Correll and published by . This book was released on 1993 with total page 230 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 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 for Cancer written by Kelly K. Hunt and published by Springer Science & Business Media. This book was released on 2007-10-26 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.

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 Innovations in Stem Cell Transplantation written by Taner Demirer and published by BoD – Books on Demand. This book was released on 2013-02-13 with total page 382 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book documents the increased number of stem cell related research, basic and clinical applications as well as views for the future. The book covers a wide range of issues related to new developments and innovations in cell-based therapies containing basic and clinical chapters from the respected authors involved in stem cell studies and research around the world. It thereby complements and extends the basic coverage of stem cells such as immunogenetics, neuron replacement therapy, cover hematopoietic stem cells, issues related to clinical problems, advanced HLA typing, alternative donor sources as well as gene therapy that employs novel methods in this field. Clearly, the treatment of various malignancies and biomedical engineering will depend heavily on stem cells, and this book is well positioned to provide comprehensive coverage of these developments.

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 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 Genomics Protocols written by Michael P. Starkey and published by Springer Science & Business Media. This book was released on 2008-02-03 with total page 537 pages. Available in PDF, EPUB and Kindle. Book excerpt: We must unashamedly admit that a large part of the motivation for editing Genomics Protocols was selfish. The possibility of assembling in a single volume a unique and comprehensive collection of complete protocols, relevant to our work and the work of our colleagues, was too good an opportunity to miss. We are pleased to report, however, that the outcome is something of use not only to those who are experienced practitioners in the genomics field, but is also valuable to the larger community of researchers who have recognized the potential of genomics research and may themselves be beginning to explore the technologies involved. Some of the techniques described in Genomics Protocols are clearly not restricted to the genomics field; indeed, a prerequisite for many procedures in this discipline is that they require an extremely high throughput, beyond the scope of the average investigator. However, what we have endeavored here to achieve is both to compile a collection of procedures concerned with geno- scale investigations and to incorporate the key components of “bottom-up” and “top-down” approaches to gene finding. The technologies described extend from those traditionally recognized as coming under the genomics umbrella, touch on proteomics (the study of the expressed protein complement of the genome), through to early therapeutic approaches utilizing the potential of genome programs via gene therapy (Chapters 27–30).

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.

Download or read book Macular Dystrophies written by Giuseppe Querques and published by Springer. This book was released on 2016-02-15 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt: ​This book provides the ophthalmologist with the most recently available data on the macular dystrophies, a group of many different inherited or sporadic eye conditions linked by a problem with photoreceptors or other structures of the central retina. Internationally recognized experts in the field present the latest evidence and discuss their own personal experiences with regard to each of the principal dystrophies as well as some very rare entities. Topics covered include molecular biology, state-of-the-art diagnostic techniques, and the newest treatment options, including still experimental therapies. Attention is also devoted to a range of issues that continue to be debated. The editors have taken care to ensure that chapters are of a uniformly high standard while not sacrificing the originality of the individual authors. Macular Dystrophies will fully acquaint the reader with both the latest research findings and the current and emerging approaches to diagnosis and treatment.

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 Molecular Diagnostics Promises and Possibilities written by Mousumi Debnath and published by Springer Science & Business Media. This book was released on 2010-01-29 with total page 527 pages. Available in PDF, EPUB and Kindle. Book excerpt: A rapid development in diverse areas of molecular biology and genetic engineering resulted in emergence of variety of tools. These tools are not only applicable to basic researches being carried out world over, but also exploited for precise detection of abnormal conditions in plants, animals and human body. Although a basic researcher is well versed with few techniques used by him/her in the laboratory, they may not be well acquainted with methodologies, which can be used to work out some of their own research problems. The picture is more blurred when the molecular diagnostic tools are to be used by physicians, scientists and technicians working in diagnostic laboratories in hospitals, industry and academic institutions. Since many of them are not trained in basics of these methods, they come across several gray areas in understanding of these tools. The accurate application of molecular diagnostic tools demands in depth understanding of the methodology for precise detection of the abnormal condition of living body. To meet the requirements of a good book on molecular diagnostics of students, physicians, scientists working in agricultural, veterinary, medical and pharmaceutical sciences, it needs to expose the reader lucidly to: Give basic science behind commonly used tools in diagnostics Expose the readers to detailed applications of these tools and Make them aware the availability of such diagnostic tools The book will attract additional audience of pathologists, medical microbiologists, pharmaceutical sciences, agricultural scientists and veterinary doctors if the following topics are incorporated at appropriate places in Unit II or separately as a part of Unit-III in the book. Molecular diagnosis of diseases in agricultural crops Molecular diagnosis of veterinary diseases. Molecular epidemiology, which helps to differentiate various epidemic strains and sources of disease outbreaks. Even in different units of the same hospital, the infections could be by different strains of the same species and the information becomes valuable for infection control strategies. Drug resistance is a growing problem for bacterial, fungal and parasitic microbes and the molecular biology tools can help to detect the drug resistance genes without the cultivation and in vitro sensitivity testing. Molecular diagnostics offers faster help in the selection of the proper antibiotic for the treatment of tuberculosis, which is a major problem of the in the developing world. The conventional culture and drug sensitivity testing of tuberculosis bacilli is laborious and time consuming, whereas molecular diagnosis offers rapid drug resistant gene detection even from direct clinical samples. The same approach for HIV, malaria and many more diseases needs to be considered. Molecular diagnostics in the detection of diseases during foetal life is an upcoming area in the foetal medicine in case of genetic abnormalities and infectious like TORCH complex etc. The book will be equally useful to students, scientists and professionals working in the field of molecular diagnostics.

Download or read book Biopharmaceutical Drug Design and Development written by Susanna Wu-Pong and published by Springer Science & Business Media. This book was released on 2010-01-11 with total page 374 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a comprehensive examination of the newest biopharmaceutical drugs. Among the drugs discussed are ones in the categories of monoclonal antibodies for in-vivo use, cytokines, growth factors, enzymes, immunomodulators, thrombolytics, and immonotherapies including vaccines. Additionally, the volume examines new and emerging technologies, and contains a review of the Human Genome Project.