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Pioneering Stem Cell Gene Therapy Trials
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Book Synopsis Pioneering Stem Cell/Gene Therapy Trials by : Roger Bertolotti
Download or read book Pioneering Stem Cell/Gene Therapy Trials written by Roger Bertolotti and published by CRC Press. This book was released on 2003-10-15 with total page 330 pages. Available in PDF, EPUB and Kindle. Book excerpt: The first unequivocal success for Gene Therapy was reported in April 2000 for X-SCID patients. Pioneering stem cell/gene therapy clinical trials are the focus of this book. Therapy successes such as the X-SCID trial and improved ADA-SCID ones are presented together with pioneering angio/vasculogenic clinical trials mediated either by transient gene
Book Synopsis Autologous and Cancer Stem Cell Gene Therapy by : Roger Bertolotti
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.
Author :National Academies of Sciences, Engineering, and Medicine Publisher :National Academies Press ISBN 13 :0309672988 Total Pages :127 pages Book Rating :4.3/5 (96 download)
Book Synopsis Exploring Novel Clinical Trial Designs for Gene-Based Therapies by : National Academies of Sciences, Engineering, and Medicine
Download or read book Exploring Novel Clinical Trial Designs for Gene-Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Book Synopsis Revolutionary Therapies: How The California Stem Cell Program Saved Lives, Eased Suffering - And Changed The Face Of Medicine Forever by : Don C Reed
Download or read book Revolutionary Therapies: How The California Stem Cell Program Saved Lives, Eased Suffering - And Changed The Face Of Medicine Forever written by Don C Reed and published by World Scientific. This book was released on 2020-03-04 with total page 369 pages. Available in PDF, EPUB and Kindle. Book excerpt: For author Don C Reed, father of a paralyzed son, the California Institute for Regenerative Medicine (CIRM) is the greatest medical advance since penicillin.REVOLUTIONARY THERAPIES is Reed's third book about the $3 billion stem cell program.Voted into law in November 2004, CIRM is now running out of money.Should its funding be renewed? Thereby hangs a tale, or rather several dozen of them, for each of the book's 71 short chapters is framed by a yarn or vignette.The factual background is accurate, vetted by the scientists, but Reed's goal is clearly both entertainment and education.A favorite example is a little girl named Evie, imprisoned in a plastic bubble: her body's immune system did not work, and she would die outside. She joined a CIRM clinical trial ... Imagine how Evie's parents felt — when she got well.Some stories are comical, like 'How Stem Cell Research Saved My Car'; others surprising, like the comparison between politics and the giant crocodile Gustave; others are tragic or inspiring: but all point to this: More than 100 million Americans suffer chronic disease, causing mountains of medical debt — and the only way to reduce that expense ($3 trillion last year) — is cure.Related Link(s)
Book Synopsis Stem Cell Therapy by : Erik V. Greer
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.
Book Synopsis How Adult Stem Cell Therapies Can Save Your Life by : Bernard van Zyl
Download or read book How Adult Stem Cell Therapies Can Save Your Life written by Bernard van Zyl and published by iUniverse. This book was released on 2009-03-20 with total page 172 pages. Available in PDF, EPUB and Kindle. Book excerpt: Laced with hope and promise for sufferers of seemingly incurable illnesses, How Adult Stem Cell Therapies Can Save Your Life: Medicines Best Kept Secret Saved My Life reveals that non-controversial adult stem cell therapies are already available for those with serious diseases and injuries. This book is a practical guide to those seeking adult stem cell treatments with step-by-step instructions on how to use the Internet to locate suitable FDA approved clinical trials for almost all major infirmities Seven years ago, van Zyl suffered cardiac arrest; his heart literally stopped until doctors shocked his chest. Over the next ten months, after receiving bypass surgery, an angioplasty, and entering a cardiac rehabilitation program, van Zyls heart was still deteriorating. Turned down by two heart transplant centers, van Zyls only hope lay in the Food and Drug Administration (FDA), or more specifically, a new FDA-approved clinical trial for adult stem cell therapies. One of 24 patients selected with heart failure, van Zyl entered the aggressive treatment group in 2004, where adult stem cells were harvested from van Zyls own body and injected directly into his failing heart. The results were astonishing. Now 75, van Zyl lives a healthy, vigorous life. How Adult Stem Cell Therapies Can Save Your Life offers a plethora of information on the latest adult stem cell treatments for over a dozen diseases and conditions. While much has been made of the medical viability and ethical concerns of other types of stem cell therapies, van Zyl is living proof that adult stem cell therapies can save lives.
Book Synopsis Mesenchymal Stromal Cells by : Hematti Peiman
Download or read book Mesenchymal Stromal Cells written by Hematti Peiman and published by Academic Press. This book was released on 2016-08-11 with total page 362 pages. Available in PDF, EPUB and Kindle. Book excerpt: Mesenchymal Stromal Cells: Translational Pathways to Clinical Adoption provides the latest information on the necessary steps for successful production of stem cells for a clinical trial. Written by professionals with hands-on experience in bringing MSC therapies to the clinic, and building on the biology and mechanisms of action, this unique book covers the development and production of clinical-grade products that are suitable for use in humans. From design of a cell production facility, to obtaining regulatory approval and reimbursement issues, it is a useful guide for researchers and administrators across biomedical research. Provides methodologies for clinical MSC production, from designing a facility, to post-market approval Includes real-life examples of MSC production in academic centers and MSC production for biopharmaceutical clinical trials Offers a unique perspective on the clinical aspects of MSC studies Presents the principles of clinical trials that can be applied to the production of various cell therapies
Book Synopsis Gene Therapy for HIV by : Gerhard Bauer
Download or read book Gene Therapy for HIV written by Gerhard Bauer and published by Springer Science & Business Media. This book was released on 2014-02-07 with total page 77 pages. Available in PDF, EPUB and Kindle. Book excerpt: This Brief describes the concept and realization of gene therapy for HIV from the unique historic perspective and insight of two pioneers of the clinical applications of stem cell gene therapy for HIV. Gerhard Bauer applied ribozyme-anti-HIV and other vectors to manufacture clinical grade, HIV-resistant hematopoietic stem cells for the first patients that received stem cell gene therapy for HIV, including the first child in the world and the first fully marrow-ablated HIV infected patient. Joseph Anderson developed the most recent and most potent combination anti-HIV lentiviral vectors and pluripotent stem cell applications for HIV gene therapy and tested these in the appropriate in vitro and vivo models, paving the way for novel HIV gene therapy approaches to possibly cure patients. In Gene Therapy for HIV, Bauer and Anderson discuss the unique aspects of this therapy, including its limitations and proper safety precautions and outline a path for a possible functional cure for HIV using stem cell gene therapy based on a cure already achieved with a bone marrow stem cell transplantation performed in Germany using donor stem cells with a naturally arising CCR5 mutation. In addition, the Brief provides a thorough and methodical explanation of the basics of gene therapy, gene therapy vector development, in vitro and in vivo models for HIV gene therapy and clinical applications of HIV gene therapy, including Good Manufacturing Practices.
Book Synopsis Novel Perspectives of Stem Cell Manufacturing and Therapies by : Diana Kitala
Download or read book Novel Perspectives of Stem Cell Manufacturing and Therapies written by Diana Kitala and published by BoD – Books on Demand. This book was released on 2021-04-14 with total page 180 pages. Available in PDF, EPUB and Kindle. Book excerpt: In the 1950s, Nobel Prize winner Dr. E. Donnall Thomas was the first to successfully transplant hematopoietic stem cells. Since then, studies on stem cells have evolved and expanded worldwide. There are more than 650,000 scientific publications on stem cells and more than 8000 stem cell clinical trials. This book summarizes types of stem cells, key studies, ongoing trials, and future perspectives. It also includes ethical, formal, and legal aspects to give the reader a comprehensive view of the field.
Book Synopsis Stem Cell and Gene-Based Therapy by : Alexander Battler
Download or read book Stem Cell and Gene-Based Therapy written by Alexander Battler and published by Springer Science & Business Media. This book was released on 2007-06-26 with total page 460 pages. Available in PDF, EPUB and Kindle. Book excerpt: Regenerative medicine – stem cell and gene-based therapy – offers a new approach for restoring function of damaged organs and tissues. This is the first book to cover the major new aspects and field of regenerative medicine. This title is therefore a timely addition to the literature. It brings together the major approaches to regenerative medicine in one text, which ensures that techniques learnt in one discipline are disseminated across other areas of medicine.
Book Synopsis Mesenchymal Stem Cell Therapy by : Lucas G. Chase
Download or read book Mesenchymal Stem Cell Therapy written by Lucas G. Chase and published by Springer Science & Business Media. This book was released on 2012-12-12 with total page 458 pages. Available in PDF, EPUB and Kindle. Book excerpt: Over the past decade, significant efforts have been made to develop stem cell-based therapies for difficult to treat diseases. Multipotent mesenchymal stromal cells, also referred to as mesenchymal stem cells (MSCs), appear to hold great promise in regards to a regenerative cell-based therapy for the treatment of these diseases. Currently, more than 200 clinical trials are underway worldwide exploring the use of MSCs for the treatment of a wide range of disorders including bone, cartilage and tendon damage, myocardial infarction, graft-versus-host disease, Crohn’s disease, diabetes, multiple sclerosis, critical limb ischemia and many others. MSCs were first identified by Friendenstein and colleagues as an adherent stromal cell population within the bone marrow with the ability to form clonogenic colonies in vitro. In regards to the basic biology associated with MSCs, there has been tremendous progress towards understanding this cell population’s phenotype and function from a range of tissue sources. Despite enormous progress and an overall increased understanding of MSCs at the molecular and cellular level, several critical questions remain to be answered in regards to the use of these cells in therapeutic applications. Clinically, both autologous and allogenic approaches for the transplantation of MSCs are being explored. Several of the processing steps needed for the clinical application of MSCs, including isolation from various tissues, scalable in vitro expansion, cell banking, dose preparation, quality control parameters, delivery methods and numerous others are being extensively studied. Despite a significant number of ongoing clinical trials, none of the current therapeutic approaches have, at this point, become a standard of care treatment. Although exceptionally promising, the clinical translation of MSC-based therapies is still a work in progress. The extensive number of ongoing clinical trials is expected to provide a clearer path forward for the realization and implementation of MSCs in regenerative medicine. Towards this end, reviews of current clinical trial results and discussions of relevant topics association with the clinical application of MSCs are compiled in this book from some of the leading researchers in this exciting and rapidly advancing field. Although not absolutely all-inclusive, we hope the chapters within this book can promote and enable a better understanding of the translation of MSCs from bench-to-bedside and inspire researchers to further explore this promising and quickly evolving field.
Book Synopsis Advances in Stem Cell Therapy by : Nagwa El-Badri
Download or read book Advances in Stem Cell Therapy written by Nagwa El-Badri and published by Humana Press. This book was released on 2016-11-18 with total page 310 pages. Available in PDF, EPUB and Kindle. Book excerpt: The book reviews the main approaches for generation of differentiated cells from various types of stem cells, including embryonic, placental and cord blood stem cells through marrow, adipose tissue and dental pulp. The book starts with an overview of experimental protocols applied to generate insulin secreting cells, neural cells, heart cells, and other tissue specific cells ex vivo and in experimental animals. This is followed by exhaustive review of clinical trials in these pathologies. It continues with a comparison of the merits of successful transplantation in humans versus animal experimentation, and highlights the most promising clinical applications in the field. Special chapters are devoted to the topic of tissue engineering and modern synthetic and biological scaffolds. It is essential reading for scientists and researchers in tissue engineering and stem cell research as well as clinicians who are involved in developing or testing stem cell therapies.
Book Synopsis Progenitor and Stem Cell Technologies and Therapies by : Anthony Atala
Download or read book Progenitor and Stem Cell Technologies and Therapies written by Anthony Atala and published by Elsevier. This book was released on 2012-03-15 with total page 561 pages. Available in PDF, EPUB and Kindle. Book excerpt: Progenitor and stem cells have the ability to renew themselves and change into a variety of specialised types, making them ideal materials for therapy and regenerative medicine. Progenitor and stem cell technologies and therapies reviews the range of progenitor and stem cells available and their therapeutic application. Part one reviews basic principles for the culture of stem cells before discussing technologies for particular cell types. These include human embryonic, induced pluripotent, amniotic and placental, cord and multipotent stem cells. Part two discusses wider issues such as intellectual property, regulation and commercialisation of stem cell technologies and therapies. The final part of the book considers the therapeutic use of stem and progenitor cells. Chapters review the use of adipose tissue-derived stem cells, umbilical cord blood (UCB) stem cells, bone marrow, auditory and oral cavity stem cells. Other chapters cover the use of stem cells in therapies in various clinical areas, including lung, cartilage, urologic, nerve and cardiac repair. With its distinguished editor and international team of contributors, Progenitor and stem cell technologies and therapies is a standard reference for both those researching in cell and tissue biology and engineering as well as medical practitioners investigating the therapeutic use of this important technology. Reviews the range of progenitor and stem cells available and outlines their therapeutic application Examines the basic principles for the culture of stem cells before discussing technologies for particular cell types, including human embryonic, induced pluripotent, amniotic and placental, cord and multipotent stem cells Includes a discussion of wider issues such as intellectual property, regulation and commercialisation of stem cell technologies and therapies
Book Synopsis A Roadmap to Nonhematopoietic Stem Cell-Based Therapeutics by : Xiao-Dong Chen
Download or read book A Roadmap to Nonhematopoietic Stem Cell-Based Therapeutics written by Xiao-Dong Chen and published by Academic Press. This book was released on 2018-08-31 with total page 544 pages. Available in PDF, EPUB and Kindle. Book excerpt: A Roadmap to Non-hematopoietic Stem Cell-Based Therapeutics: From the Bench to the Clinic is a resource that provides an overview of the principles of stem cell therapy, the promises and challenges of using stem cells for treating various clinical conditions, and future perspectives. The overall goal is to facilitate the translation of basic research on stem cells to clinical applications. The properties of stem cells from various sources are reviewed and the advantages and disadvantages of each for clinical use are discussed. Modifying stem cell properties through preconditioning strategies using physical, chemical, genetic, and molecular manipulation to improve cell survival, increase cell differentiation potential, enhance production of paracrine factors, and facilitate homing to the site of injury or disease upon transplantation are reviewed. Various routes of stem cell administration and dosing, and the duration of effects, are explored. Individual chapters are written by experts in the field and focus on the use of stem cells in treating various degenerative diseases, autoimmune diseases, wound healing, cardiovascular disease, spinal cord injury, oral and dental diseases, and skeletal disorders. Finally, experts in the regulatory arena discuss mechanisms used in different countries for approving the use of stem cells to treat diseases and many common issues that are typically encountered while seeking approval for this class of therapeutic agent. Offers advanced students, as well as new researchers, an overview of the principles of stem cell therapy Discusses a wide array of pressing clinical issues with stem cell-based therapies so that new ideas in the laboratory can be efficiently translated to the clinic through better designed clinical trials Helps clarify current regulatory mechanisms so that the safe use of stem cells for treating a variety of diseases can move forward Fosters cross-disciplinary dialogue between research scientists and physicians to accelerate the safe implementation of efficacious cell therapies
Book Synopsis Safety, Efficacy and Mechanisms of Action of Mesenchymal Stem Cell Therapies by : Guido Moll
Download or read book Safety, Efficacy and Mechanisms of Action of Mesenchymal Stem Cell Therapies written by Guido Moll and published by Frontiers Media SA. This book was released on 2020-07-24 with total page 296 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Regenerative Medicine - from Protocol to Patient by : Gustav Steinhoff
Download or read book Regenerative Medicine - from Protocol to Patient written by Gustav Steinhoff and published by Springer. This book was released on 2016-06-14 with total page 396 pages. Available in PDF, EPUB and Kindle. Book excerpt: Regenerative medicine is the main field of groundbreaking medical development and therapy using knowledge from developmental and stem cell biology as well as advanced molecular and cellular techniques. This collection of volumes on Regenerative Medicine: From Protocol to Patient, aims to explain the scientific knowledge and emerging technology as well as the clinical application in different organ systems and diseases. International leading experts from all over the world describe the latest scientific and clinical knowledge of the field of regenerative medicine. The process of translating science of laboratory protocols into therapies is explained in sections on regulatory, ethical and industrial issues. This collection is organized into five volumes: (1) Biology of Tissue Regeneration, (2) Stem Cell Science and Technology, (3) Tissue Engineering, Biomaterials and Nanotechnology, (4) Regenerative Therapies I, and (5) Regenerative Therapies II. The textbook gives the student, the researcher, the health care professional, the physician and the patient a complete survey on the current scientific basis, therapeutical protocols, clinical translation and practiced therapies in regenerative medicine. Volume 5 contains clinical science and translation surveys on the circulatory system, visceral, musculoskeletal and skin. The state-of-the-art descriptions involve concepts for clinical diagnosis, stem cell and gene therapy, biomaterials for tissue replacement and pharmacological/biomolecule treatment strategies.
Book Synopsis Frontiers in Stem Cell and Regenerative Medicine Research by : Atta-ur-Rahman Rahman
Download or read book Frontiers in Stem Cell and Regenerative Medicine Research written by Atta-ur-Rahman Rahman and published by Bentham Science Publishers. This book was released on 2017-07-06 with total page 264 pages. Available in PDF, EPUB and Kindle. Book excerpt: Stem cell and regenerative medicine research is a hot area of research which promises to change the face of medicine as it will be practiced in the years to come. Challenges in the 21st century to combat diseases such as cancer, Alzheimer and related diseases may well be addressed employing stem cell therapies and tissue regeneration. Frontiers in Stem Cell and Regenerative Medicine Research is essential reading for researchers seeking updates in stem cell therapeutics and regenerative medicine. The sixth volume of this series features reviews on roles of mesenchymal stem cells in cartilage regeneration and bone regeneration, liver regeneration, cardiogenesis, cardiomyocyte differentiation, and regenerative therapy for neurodegenerative disorders.
