Download or read book In Vivo Gene Transfer Into Mobilized Hematopoietic Stem Cells written by Maximilian Richter and published by . This book was released on 2017 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Stem Cells from Cord Blood in Utero Stem Cell Development and Transplantation Inclusive Gene Therapy written by W. Holzgreve and published by Springer Science & Business Media. This book was released on 2013-03-09 with total page 265 pages. Available in PDF, EPUB and Kindle. Book excerpt: The title "Stem Cells from Cord Blood, In Utero Stem Cell Develop ment, and Transplantation-Inclusive Gene Therapy" suggests that more than one topic is combined in one workshop. Indeed, at first glance the recovery of stem cells from cord blood has to be seen as separate from the attempts to achieve effective in utero therapy by stem cell trans plantation, because the first issue deals with an innovative stem cell source as an alternative to bone marrow, which is already spreading rapidly in medical practice, whereas the second topic is still strictly ex perimental and only investigated in medical centers with the appropri ate background. It is, however, not only justified, but helpful to com bine the two topics in one workshop and consequently to cover them in the same volume of the Ernst Schering Research Foundation Work shop series, because they are intimately related and both based on the new insights into the biology of stem cells. Professor Werner Arber, the Nobel Laureate from the University of Basel, pointed out in his In- Professor Dr. W. Holzgreve VI Preface The participants of the workshop troductory Lecture that our understanding of hematopoietic stem cells as descendents of totipotent cells and our current approaches to using them in post-and prenatal therapy have been furthered significantly by genetic engineering technologies which are "artificial contributions to the process of biologic evolution".

Download or read book Ex Vivo Cell Therapy written by Klaus Schindhelm and published by Academic Press. This book was released on 1999 with total page 392 pages. Available in PDF, EPUB and Kindle. Book excerpt: R.E. Nordon and K. Schindhelm, Introduction. -- L. Robb, A.G. Elefanty, and C.G. Begley, Transcriptional Control of Hematopoieses. -- R. Starr and N.A. Nicola, Cell Signaling by Hemopoietic Growth Factor Receptors. -- P.J. Simmons, D.N. Haylock, and J.-P. Lévesque, Influence of Cytokines and Adhesion Molecules on Hematopoietic Stem Cell Development. -- P.A. Rowlings, Allogeneic Hematopoietic Stem Cell Transplantation. -- U. Hahn and L.B. To, Autologous Stem Cell Transplantation. -- M.R. Vowels, Cord Blood Stem Cell Transplantation. -- S.R. Riddell, E.H. Warren, D. Lewinsohn, C. Yee, and P.D. Greenberg, Reconstitution of Immunity by Adoptive Immunotherapy with T Cells. -- L.Q. Sun, M. Miller, and G. Symonds, Exogenous Gene Transfer into Lymphoid and Hematopoietic Progenitor Cells. -- C. Dowding, T. Leemhuis, A. Jakubowski, and C. Reading, Process Development for Ex Vivo Cell Therapy. -- R.E. Nordon and K. Schindhelm, Cell Separation. -- P.W. Zandstra, C.J. Eaves, and J.M. Piret, Environ ...

Download or read book Gene Transfer Into Hemopoietic Stem Cells written by Markus Peter Wilhelmus Einerhand and published by . This book was released on 1992 with total page 108 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 Marrow Protection written by Joseph R. Bertino and published by Karger Medical and Scientific Publishers. This book was released on 1999-01-01 with total page 193 pages. Available in PDF, EPUB and Kindle. Book excerpt: Authored by experts in the field, this volume provides a comprehensive review of the problems and opportunities in gene therapy focusing on the use of various drug resistance genes to provide protection of hematopoietic cells against drug toxicity. General topics include basic principles of gene transfer, optimizing conditions for gene transfer in hematopoietic cells, and gene transfer in the nonmyeloablated host. Specific chapters describe constructions and preclinical studies of specific genes that impart resistance to anticancer drugs when expressed in hematopoietic cells including MDR-1, mutated forms of human methylguanine-DNA-methyltranferase, mutant forms of dihydrofolate reductase and thymidylate synthase, dihydropyrimidine dehydrogenase and cytidine deaminase. For successful transfer of drug resistance genes, the possibility that they may be used as selectable markers and that marrow ablation may not be required are important considerations. This book is especially timely, as clinical trials with these drug resistance genes are expected to increase markedly in the next few years. It should appeal to all interested in stem cell biology and gene therapy, as well as clinicians who look forward to using this technology in their practice.

Download or read book Hematopoietic Stem Cells written by Louis M. Pelus and published by Springer Nature. This book was released on 2022-10-18 with total page 284 pages. Available in PDF, EPUB and Kindle. Book excerpt: This detailed volume collects updates on the technical advances in hematopoietic stem cell research and incorporates new techniques focused on the molecular/genetic, cellular, and whole organism levels. Exploring methods that apply stress to hematopoiesis, the book also contains chapters focused on better understanding the role of hematopoietic niches and their cellular components, as well as in vivo models that test and quantitate stem cell function and are key to further development of therapeutic applications. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and up-to-date, Hematopoietic Stem Cells: Methods and Protocols serves as a valued addition to laboratories focused on understanding hematopoietic stem cell biology and the therapeutic advances that can be derived from it.

Download or read book Autologous and Cancer Stem Cell Gene Therapy written by Roger Bertolotti and published by World Scientific. This book was released on 2008 with total page 489 pages. Available in PDF, EPUB and Kindle. Book excerpt: Stem cells provide for life-long cell replacement in tissues and organs, and have inherent homing abilities that are critical in therapeutic applications. Stem cells are also the driving force of cancer where genetic/epigenetic alterations culminate in tumorigenesis either in tissue stem cells or in some of their derivatives. As a rare subset of the tumor, cancer stem cells are the only drive of tumor initiation/propagation. Autologous and cancer stem cells are thus the key targets of 1) long-term and transient-regenerative/epigenetic gene therapy and 2) of recurrence-free anticancer therapy, respectively. While cancer stem cell gene therapy still needs time to accomplish, autologous stem cells have been instrumental in the first unequivocal successes for gene therapy whereby ex vivo retrovirally corrected hematopoietic stem cells have been returned to the patients.This timely book presents 1) the aforementioned stem cell gene therapy achievements that rely on random-integration of therapeutic transgenes into host chromosomes and 2) emerging experimental approaches aimed at eliminating random-integration oncogenic hazards through site-specific integration or gene targeting. Breakthrough endonuclease-boosted gene targeting for gene correction (inherited diseases) or targeted integration of therapeutic transgenes (other disorders) culminating in an efficiency compatible with clinical trials is one of the highlights of the book. Highlights also include the pioneering transplantation of adult pluripotent stem cells as a substitute for tissue-specific stem cells, thereby pinpointing the invaluable potential for stem cell gene therapy applications of autologous cells able to contribute to all three germ layers. Stem cell gene therapy is thus discussed in terms of 1) magnifying stem cell therapeutic homing through transient regenerative gene therapy and 2) of tackling most pathologies (including mitochondrial DNA diseases and ageing disorders) through stem cell repopulation dynamics into appropriate niches (long-term engraftment) and tissues (cell turn-over). Regarding cancer stem cell gene therapy, focus is on both the increasing number of identified tissue-specific cancer stem cells as the ultimate therapeutic targets and on the development of armed stem cells as tumor-homing vectors for targeted anticancer therapy.

Download or read book Advances in Hematopoietic Stem Cell Transplantation and Molecular Therapy written by Rainer Haas and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 159 pages. Available in PDF, EPUB and Kindle. Book excerpt: Blood stem cell transplantation has become an established therapeutic option in the treatment of malignant diseases. At the same time molecular therapeutic approaches (i.e. gene therapy) are promising alternatives to the classical treatment of cancer. The proceedings give an overview of the biology of hematopoietic progenitor cells, peripheral stem cell transplantation in patients with hematological malignancies, recent developments in molecular diagnosis and gene therapeutic concepts, and show that successful future developments require cooperative research connecting basic science with clinical application.

Download or read book Advances in Allogeneic Hematopoietic Stem Cell Transplantation written by Richard K. Burt and published by Springer. This book was released on 2012-11-28 with total page 419 pages. Available in PDF, EPUB and Kindle. Book excerpt: The field of hematopoietic stem cell transplantation is rapidly evolving. Realization that hematopoietic stem cells give rise to the immune compartment has resulted in clinical trials of hematopoietic stem cell transplantation for patients with autoimmune diseases. Allogeneic hematopoietic transplants are a form of adoptive immunotherapy resulting in beneficial graft versus tumor effects. Large numbers of hematopoietic cells can be collected with ease. Therefore, a renewable source of cells for ex vivo genetic manipulations is readily available. Multiple trials combining hematopoietic transplants and gene therapy are in progress. One such application is the infusion of allogeneic lymphocytes containing a suicide gene to abort graft versus host disease. Hematopoietic stem cell transplantation is in reality the clinical and practical application of cellular therapy. Hematopoietic transplant physicians are by design or by practical application evolving into cell and gene therapy specialists. The excitement and enthusiasm in hematopoietic transplantation is that it offers a door to the future. A future not of drugs or titrating poisonous chemotherapy but rather of cellular and gene therapy. 1 ALLOGENEIC PERIPHERAL BLOOD STEM CELL TRANSPLANTATION FOR HEMATOLOGIC DISEASES Martin Korbling University o/Texas MD. Anderson Cancer Center, Houston, Texas 77030 INTRODUCTION Circulating hematopoietic stem cells have emerged as an alternative to bone marrow (BM) stem cells for allografting. For many years the reconstitutive potential of circulating stem cells was questioned; peripheral blood stem cells (PBSC) were even characterized a waste product (1).

Download or read book Assessment and Improvement of Gene Transfer Into Human Hematopoietic Stem Cells written by Dimitri Anthonius Breems and published by . This book was released on 1997 with total page 164 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Cell Therapy written by George Morstyn and published by Cambridge University Press. This book was released on 1996-03-29 with total page 656 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cell therapy is a rapidly developing area, drawing on cell biology, molecular biology, virology, immunology, cell quantitation techniques and biomedical engineering. It has potential in many clinical settings, in the treatment of cancer and other diseases. This volume in the series Cancer: Clinical Science in Practice examines the current state and future prospects of cell therapy, which seems likely to have an even more profound impact on health care than did the production of proteins by recombinant DNA technology. The coverage is broad, including the scientific principles of haematopoietic cell therapy, the technology of cell collection and preparation, current and likely future clinical applications of cell therapy, and the principles and practice of cellular immunotherapy. Up-to-date and authoritative, volumes in this series are intended for a wide audience of clinicians and researchers with an interest in the applications of biomedical science to the understanding and management of cancer.

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 New Methods for Lentiviral Based Hematopoietic Stem Cell Gene Therapy written by Katelyn Masiuk and published by . This book was released on 2019 with total page 101 pages. Available in PDF, EPUB and Kindle. Book excerpt: Hematopoietic stem cell (HSC) transplant with gene therapy has recently emerged as a successful clinical treatment of a number of previously incurable genetic blood diseases. This approach aims to permanently fix genetic defects in HSCs, a rare and specialized type of cell with the unique ability to regenerate the entire blood system throughout a patient's lifetime. In this approach, bone marrow (BM) or mobilized peripheral blood (mPB) is collected from a patient, enriched for HSCs, transduced with an engineered lentiviral vector (LV) encoding the correct genetic sequence, and transplanted back into the patient. After transplant, modified HSCs engraft in the BM and produce healthy blood cells throughout the patient's lifetime. While the last decade of research has yielded major advances including successful Phase I/II gene therapy clinical trials, clinical and commercial scaling of this technology to a broader range of patients and diseases has revealed a number of hurdles. One major limitation is the great expense and difficulty of producing clinical-grade LV, which I address in Chapters 2 and 3 by presenting two methods that improve the efficiency of LV transduction of HSC. In Chapter 4, I demonstrate the successful application of a new LV gene therapy for an autoimmune blood disease. Chapter 2 presents a method to enhance the enrichment of HSCs from the heterogeneous cell population obtained from the collection of bone marrow cells, addressing a critical limitation in creating cost-effective, clinical-grade LV vector. This method utilizes immunomagnetic beads to purify CD34+CD38- cells, a population highly enriched for HSCs beyond standard CD34+ selection. Using immune-deficient xenograft models, we demonstrate that enrichment of CD34+CD38- cells reduces gene therapy culture scale and lentiviral vector requirements by ~10-fold while still maintaining the long-term gene-marked engraftment required for clinical benefit. Therefore, this strategy represents an easily translatable method which can conserve valuable clinical grade LV preparations and could lower the cost per patient, or allow for the treatment of a greater number of patients. Chapter 3 presents a method to further improve HSC transduction efficiency with the use of two compounds: Prostaglandin E2 (PGE2) and poloxamer synperonic F108 (PS-F108). While transduction enhancement with each individual compound has previously been reported, the combination of these compounds leads to a synergistic and marked improvement in LV transduction of HSCs using a globin LV. Remarkably, this synergistic combination achieved a 6-fold improvement in gene transfer to long-term engrafting HSCs while using a LV dose 10-fold lower than the dose in our current clinical protocol. Thus this strategy has two major advantages: it reduces the amount of viral particles required to transduce HSCs, and also allows for better gene transfer and ultimate globin transgene expression, which is critical to improving clinical efficacy. Finally, chapter 4 demonstrates the effectiveness of a newly engineered LV for the treatment of a severe form of genetic autoimmunity called IPEX syndrome. IPEX is caused by mutations in FoxP3, the key lineage-determining transcription factor required for the development and function of regulatory T cells (Treg cells). We developed a new LV using endogenous human FOXP3 regulatory elements to restore FoxP3 expression in a developmentally appropriate manner. We use this LV to transduce HSCs and restore functional Treg development in a mouse model of FoxP3 deficiency and successfully rescue autoimmune defects associated with this phenotype. These findings demonstrate preclinical efficacy for the treatment of IPEX patients by autologous HSC transplant and may provide further insight into new cell therapies for autoimmunity. Collectively, the work described here advances the field of gene therapy by improving the efficiency of the manufacturing process and expanding the range of diseases which can be treated by this method.

Download or read book Stem Cell Therapy written by Erik V. Greer and published by Nova Publishers. This book was released on 2006 with total page 248 pages. Available in PDF, EPUB and Kindle. Book excerpt: Among the many applications of stem cell research are nervous system diseases, diabetes, heart disease, auto-immune diseases as well as Parkinson's disease, end-stage kidney disease, liver failure, cancer, spinal cord injury, multiple sclerosis, Parkinson's disease, and Alzheimer's disease. Stem cells are self-renewing, unspecialised cells that can give rise to multiple types all of specialised cells of the body. Stem cell research also involves complex ethical and legal considerations since they involve adult, foetal tissue and embryonic sources. This new book brings together leading research from throughout the world in this frontier field.

Download or read book Hematopoietic Stem Cells written by Christa Müller-Sieburg and published by Springer. This book was released on 1992 with total page 278 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Translating Gene Therapy to the Clinic written by Jeffrey Laurence and published by Academic Press. This book was released on 2014-11-14 with total page 347 pages. Available in PDF, EPUB and Kindle. Book excerpt: Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials