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Gene Therapy - From Laboratory To The Clinic

Gene Therapy - From Laboratory To The Clinic PDF Author: Kam M Hui
Publisher: World Scientific
ISBN: 9814502383
Category : Science
Languages : en
Pages : 289

Book Description
With advances in our understanding of the molecular biology of human diseases and the development of efficient gene transfer techniques, the treatment of such diseases as cancer and infectious disease using gene therapy has progressed from a distant prospect to a distinct possibility in a very short time. The development of gene transfer methods which are suitable for different forms of therapy has been a major topic of research over the past several years. A common goal of this research has been to achieve the efficient delivery of genes into cells. The successful implementation of gene transfer as a cure for diseases, however, will continue to require the translation of preclinical studies in gene therapy into effective clinical protocols. This volume outlines the latest developments in cancer treatment using various gene delivery systems, which include cytokine gene transfer, the delivery of anti-ras DNA by retroviral vector and the injection of allogeneic HLA DNA via liposomes. Several of these molecular approaches have recently been approved by the US FDA as human clinical trial protocols in order to assess their therapeutic efficiency and safety for cancer treatment. Further developments in recombinant DNA technology within this field should ultimately lead to dramatic improvements in the practice of medicine.

Gene Therapy - From Laboratory To The Clinic

Gene Therapy - From Laboratory To The Clinic PDF Author: Kam M Hui
Publisher: World Scientific
ISBN: 9814502383
Category : Science
Languages : en
Pages : 289

Book Description
With advances in our understanding of the molecular biology of human diseases and the development of efficient gene transfer techniques, the treatment of such diseases as cancer and infectious disease using gene therapy has progressed from a distant prospect to a distinct possibility in a very short time. The development of gene transfer methods which are suitable for different forms of therapy has been a major topic of research over the past several years. A common goal of this research has been to achieve the efficient delivery of genes into cells. The successful implementation of gene transfer as a cure for diseases, however, will continue to require the translation of preclinical studies in gene therapy into effective clinical protocols. This volume outlines the latest developments in cancer treatment using various gene delivery systems, which include cytokine gene transfer, the delivery of anti-ras DNA by retroviral vector and the injection of allogeneic HLA DNA via liposomes. Several of these molecular approaches have recently been approved by the US FDA as human clinical trial protocols in order to assess their therapeutic efficiency and safety for cancer treatment. Further developments in recombinant DNA technology within this field should ultimately lead to dramatic improvements in the practice of medicine.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127

Book Description
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.

Gene Therapy Technologies, Applications and Regulations

Gene Therapy Technologies, Applications and Regulations PDF Author: Anthony Meager
Publisher: John Wiley & Sons
ISBN:
Category : Medical
Languages : en
Pages : 450

Book Description
Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex 'gene medicines' containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book: surveys many of the current technologies for preparing vectors for use in gene therapy protocols reviews the application of gene-mediated therapies to a range of medical conditions considers the regulatory aspects of gene therapy including product quality and safety requirements appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for: good laboratory practice (GLP) conditions in the R&D laboratory large-scale production methods and good manufacturing practice (GMP) current in-process and final product testing Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.

Cellular Transplantation

Cellular Transplantation PDF Author: Craig Halberstadt
Publisher: Elsevier
ISBN: 0080469043
Category : Science
Languages : en
Pages : 696

Book Description
There have been tremendous strides in cellular transplantation in recent years, leading to accepted practice for the treatment of certain diseases, and use for many others in trial phases. The long history of cellular transplantation, or the transfer of cells from one organism or region of the body to another, has been revolutionized by advances in stem cell research, as well as developments in gene therapy. Cellular Transplants: From Lab to Clinic provides a thorough foundation of the basic science underpinning this exciting field, expert overviews of the state-of-the-art, and detailed description of clinical success stories to date, as well as insights into the road ahead. As highlighted by this timely and authoritative survey, scale-up technologies and whole organ transplantation are among the hurdles representing the next frontier. The contents are organized into four main sections, with the first covering basic biology, including transplant immunology, the use of immunosuppressive drugs, stem cell biology, and the development of donor animals for transplantation. The next part looks at peripheral and reconstructive applications, followed by a section devoted to transplantation for diseases of the central nervous system. The last part presents efforts to address the key challenges ahead, such as identifying novel transplantable cells and integrating biomaterials and nanotechnology with cell matrices. Provides detailed description of clinical trials in cell transplantation Review of current therapeutic approaches Coverage of the broad range of diseases addressed by cell therapeutics Discussion of stem cell biology and its role in transplantation

Gene Therapy Protocols

Gene Therapy Protocols PDF Author: Joseph LeDoux
Publisher: Humana Press
ISBN: 9781627039567
Category : Medical
Languages : en
Pages : 0

Book Description
In this book internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors and Design and Characterization of Gene Transfer Vectors. Readers will find a comprehensive resource of current and emerging methods for the production of viral and non-viral gene transfer vectors, as well as detailed protocols for applications in stem cell biology, cancer research and infectious disease.

Gene Therapy for Cancer

Gene Therapy for Cancer PDF Author: Kelly K. Hunt
Publisher: Springer Science & Business Media
ISBN: 159745222X
Category : Medical
Languages : en
Pages : 469

Book Description
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.

Translating Regenerative Medicine to the Clinic

Translating Regenerative Medicine to the Clinic PDF Author: Jeffrey Laurence
Publisher: Academic Press
ISBN: 0128005521
Category : Technology & Engineering
Languages : en
Pages : 356

Book Description
Translating Regenerative Medicine to the Clinic reviews the current methodological tools and experimental approaches used by leading translational researchers, discussing the uses of regenerative medicine for different disease treatment areas, including cardiovascular disease, muscle regeneration, and regeneration of the bone and skin. Pedagogically, the book concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field. It promotes cross-disciplinary communication between the sub-specialties of medicine, but remains unified in theme by emphasizing recent innovations, critical barriers to progress, the new tools that are being used to overcome them, and specific areas of research that require additional study to advance the field as a whole. Volumes in the series include Translating Gene Therapy to the Clinic, Translating Regenerative Medicine to the Clinic, Translating MicroRNAs to the Clinic, Translating Biomarkers to the Clinic, and Translating Epigenetics to the Clinic. Encompasses the latest innovations and tools being used to develop regenerative medicine in the lab and clinic Covers the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field Contains extensive pedagogical updates aiming to improve the education of translational researchers in this field Provides a transdisciplinary approach that supports cross-fertilization between different sub-specialties of medicine

Gene Therapy

Gene Therapy PDF Author: Theodore Friedmann
Publisher: CSHL Press
ISBN: 9780879694463
Category : Gene therapy
Languages : en
Pages : 156

Book Description
In 1982, a meeting of unusual influence was held at the Banbury Conference Center of Cold Spring Harbor Laboratory. After an early attempt at treating clinical disease with transferred genes had ignited public attention and scientific controversy, a group of distinguished biologists and physicians came together to assess practical progress towards gene therapy and what its future might be. The geneticist Ted Friedmann wrote a narrative account of the participants' contributions to the meeting, ending with a personal discussion of ethical issues raised by genetic technologies.His book, the first on gene therapy, was widely read but has long been unavailable. It has been reprinted with a new introduction entitled "Gene Therapy 1994", in which the author reviews the field's technical accomplishments and ethical dilemmas. Now that gene therapy has become part of the medical landscape, this volume is of interest as both a historical document and an assessment of the field's current challenges.

Gene Therapy Protocols

Gene Therapy Protocols PDF Author: Joseph LeDoux
Publisher: Humana Press
ISBN: 9781588299031
Category : Medical
Languages : en
Pages : 0

Book Description
In this book internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors and Design and Characterization of Gene Transfer Vectors. Readers will find a comprehensive resource of current and emerging methods for the production of viral and non-viral gene transfer vectors, as well as detailed protocols for applications in stem cell biology, cancer research and infectious disease.

Gene Therapy Protocols

Gene Therapy Protocols PDF Author: Joseph LeDoux
Publisher: Humana Press
ISBN: 9781627039642
Category : Medical
Languages : en
Pages : 0

Book Description
In Gene Therapy Protocols, Volumes 1 and 2, internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors (Volume 1) and Design and Characterization of Gene Transfer Vectors (Volume 2). In this second volume, readers will find a comprehensive resource of current and emerging methods for the processing and characterization of viral and non-viral gene transfer vectors.