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Translating Gene Therapy to the Clinic

Author: Jeffrey Laurence

Publisher: Academic Press

Published: 2014-11-14

Total Pages: 347

ISBN-13: 0128005645

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

Translating Regenerative Medicine to the Clinic

Author: Jeffrey Laurence

Publisher: Academic Press

Published: 2015-11-18

Total Pages: 356

ISBN-13: 0128005521

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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

Author: Kam M. Hui

Publisher: World Scientific

Published: 1994

Total Pages: 292

ISBN-13: 9789810216559

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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.

Nonviral Vectors for Gene Therapy

Author:

Publisher: Academic Press

Published: 2015-01-23

Total Pages: 297

ISBN-13: 0128024704

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The field of genetics is rapidly evolving, and new medical breakthroughs are occurring as a result of advances in our knowledge of genetics. Advances in Genetics continually publishes important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines. Includes methods for testing with ethical, legal, and social implications Critically analyzes future directions Written and edited by recognized leaders in the field

Gene Transfer and the Ethics of First-in-Human Research

Author: Jonathan Kimmelman

Publisher: Cambridge University Press

Published: 2010

Total Pages: 219

ISBN-13: 0521690846

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Examines the ethical and policy dimensions of testing novel medical interventions in human beings for the first time.

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2019-12-01

Total Pages: 93

ISBN-13: 0309495849

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On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.

Translating MicroRNAs to the Clinic

Author: Jeffrey Laurence

Publisher: Academic Press

Published: 2016-09-17

Total Pages: 392

ISBN-13: 0128005548

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Translating microRNA to the Clinic reviews the possibilities of current methodological tools and experimental approaches used by leading translational researchers. The book features the uses of micro ribonucleic acid as deployed in cancer targeting in biomarkers, diabetes, cardiovascular disease, and neurodegeneration, among many others. Pedagogically, the work concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field, promoting a cross-disciplinary communication between the sub-specialities of medicine, but in common with other books on the topic. In addition, the book emphasizes 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. Encompasses the latest innovations and tools being used to apply microRNA from lab to clinic“/li> Extensive pedagogical updates aiming to improve the education of translational researchers in this field Transdisciplinary approach aims to support cross-fertilization between different sub-specialties of medicine

Pharmaceutical Biotechnology

Author: Daan J. A. Crommelin

Publisher: CRC Press

Published: 2002-11-14

Total Pages: 456

ISBN-13: 9780415285018

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The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.

Fast Facts: Gene Therapy

Author: Roland Herzog

Publisher: Karger Medical and Scientific Publishers

Published: 2020-04-08

Total Pages: 102

ISBN-13: 3318066664

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Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

The Clinibook

Author: Odile Cohen-Haguenauer

Publisher: EDK Editions

Published: 2013-05-03T00:00:00+02:00

Total Pages: 589

ISBN-13: 2842542371

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This book provides a 2012 reference for state-of-the-art gene transfer technology and the different aspects of its clinical translation with a focus on European-based initiatives. As examples of successful outcomes, recent clinical trials are presented together with Ethical, Safety and Regulatory issues, which are discussed. The broad range of various technologies is covered whether addressing direct in vivo gene transfer like with AAV, Adeno or non-viral vectors or ex-vivo genetically engineered cells including induced pluripotent stem cells (iPS) with integrating vectors such as retrovirus, lentivirus or transposon-derived systems. The critical path to clinical implementation is covered in the second part describing currently available tools - such as molecular imaging, ex-vivo organ cultures and high-throughput technologies used for evaluation of criteria towards a go-or-no go decision to move to the clinic; in addition, utmost salient biosafety and immunotoxicology aspects are discussed. This book is ideal for postgraduates, undergraduates, scientists, clinicians, regulators and patients' advocacy groups looking for states-of-the-art information as well as emerging prospects - including gene targeting and homologous recombination - in gene transfer intended for clinical translation. Dr Odile Cohen-Haguenauer is the Coordinator of Clingene-NoE, the European Network for the Advancement of Clinical Gene Transfer and Therapy.