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 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 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 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 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 Gene Technology written by Axel R. Zander and published by Springer Science & Business Media. This book was released on 2013-06-29 with total page 534 pages. Available in PDF, EPUB and Kindle. Book excerpt: The 11 th meeting in "Modern Trends in Human Leukemia" took place from June 19 to 21, 1994 in Wilsede in the middle of the Liineburger Heide, South of Hamburg. Interwoven with the Leukemia program was the Nato-sponsored Symposium of the ASI-Series "Gene Technology in Analysis of Malignant and Inherited Human Diseases Related to Development" . The Wilsede meeting was continued on a ship of the Neva leading through lake Ladoga and lake Onega. The topics of both meetings included discussion on recent progress isolation and development of hematopoietic stem cells, genes crucial for development and diseases, methods of gene transfer, application of gene transfer; oncogenes and anti-oncogenes as targets for gene therapy; receptors and their ligands in normal development and diseases, immunology and immunotherapy, radiation biology, clinical leukemias and bone marrow transplantation. The Nato workshop concentrated not only on analysis of cell systems useful for somatic gene therapy, but also on actual themes directly related to correction of human diseases. The latter aspects emphasized themes related to biotechnology, the first part was by nature more general. We also included a few contributions that discussed perspectives for the future of gene therapy and possible relationships to evolution.

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 Primary and Stem Cells written by Uma Lakshmipathy and published by John Wiley & Sons. This book was released on 2011-10-31 with total page 314 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book describes basic cell engineering methods, emphasizing stem cell applications, and use of the genetically modified stem cells in cell therapy and drug discovery. Together, the chapters introduce and offer insights on new techniques for engineering of stem cells and the delivery of transgenes into stem cells via various viral and non-viral systems. The book offers a guide to the types of manipulations currently available to create genetically engineered stem cells that suit any investigator's purpose, whether it's basic science investigation, creation of disease models and screens, or cells for therapeutic applications.

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 Non stable Viral and Stable Non viral Gene Transfer to Hematopoietic Stem Cells written by Sarah Jane Nightingale and published by . This book was released on 2006 with total page 216 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 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 Identification and Characterization of Human Hematopoietic Stem Cells Using Gene Transfer and the Novel SCID Transplantation Assay written by André Larochelle and published by . This book was released on 1997 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: The absence of preclinical repopulation assays for primitive human hematopoietic cells has hindered the development of stem cell gene therapy. Current human gene therapy trials employ gene transfer methods optimized using surrogate in vitro colony-forming cell (CFC) and longterm culture-initiating cell (LTC-IC) assays and by inference from studies on other mammalian species. In this thesis, we report the identification and characterization of a novel human hematopoietic cell, capable of repopulating the bone marrow (BM) of nonobese diabetic mice with severe combined immunodeficiency (NOD/SCID), namely the SCID-repopulating cell (SRC). Human BM or cord blood (CB) cells were infected with retroviral vectors and subsequently transplanted into NOD/SCID mice. Whereas CFC and LTC-IC were efficiently transduced with retroviruses, SRCs were transduced rarely, establishing that SRC are biologically distinct from most CFC and LTC-IC. Moreover, characterization of the cell surface phenotype indicated that SRC are exclusively CD3$\sp+$CD38$\sp-$ in contrast to CFC and LTC-IC that are mostly found in the CD34$\sp+$CD38$\sp+$ fraction after multiparameter flow sorting, confirming the distinct character of SRC. In combination with various data obtained in the past decade, these analyses provide the strongest evidence that SRC defines a novel human hematopoietic cell that is more immature than any other cell type detected to date. The inefficient infection of SRC was consistent with the low level of gene marking reported in primates and human gene therapy trials. This inability to infect the most primitive human repopulating cells, in contrast to most CFC and LTC-IC, highlights the need for appropriate pre-clinical models that will predict the outcomes of human clinical trials. As described in chapter 3 of this thesis, primitive BM cells from $\beta$-thalassemia major and sickle cell anemia (SCA) patients can engraft and proliferate in the BM of immune-deficient mice. The BM of transplanted mice contained the entire erythroid lineage from BFU-E to mature erythrocytes expressing human $\gamma$-, $\beta$- or $\rm\beta\sp{s}$-globin as in the original donor. Moreover, human erythroid cells from mice transplanted with SCA bone marrow showed characteristic sickling under reducing conditions in an in vitro assay. This system can thus be used to evaluate gene transfer efficiency into primitive human cells, longevity of expression, expression in the appropriate lineage, and correction of the disease phenotype.

Download or read book Oversight and Review of Clinical Gene Transfer Protocols written by Institute of Medicine and published by National Academies Press. This book was released on 2014-03-27 with total page 78 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

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 Studies of in Utero Transplantation and Gene Transfer in Murine Fetal Liver Hematopoietic Stem Cells written by Margret L. Casal and published by . This book was released on 1999 with total page 147 pages. Available in PDF, EPUB and Kindle. Book excerpt: