Translating Gene Therapy To The Clinic

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Translating Gene Therapy to the Clinic

Jeffrey Laurence,Michael Franklin
Translating Gene Therapy to the Clinic Book PDF

Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Page : 347 pages
File Size : 47,8 Mb
Release : 2014-11-14
Category : Science
ISBN : 9780128005644

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Translating Gene Therapy to the Clinic by Jeffrey Laurence,Michael Franklin Pdf

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

Jeffrey Laurence,Pedro Baptista,Anthony Atala
Translating Regenerative Medicine to the Clinic Book PDF

Author : Jeffrey Laurence,Pedro Baptista,Anthony Atala
Publisher : Academic Press
Page : 356 pages
File Size : 48,8 Mb
Release : 2015-11-18
Category : Technology & Engineering
ISBN : 9780128005521

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Translating Regenerative Medicine to the Clinic by Jeffrey Laurence,Pedro Baptista,Anthony Atala Pdf

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 - From Laboratory To The Clinic

Kam M Hui
Gene Therapy From Laboratory To The Clinic Book PDF

Author : Kam M Hui
Publisher : World Scientific
Page : 289 pages
File Size : 51,5 Mb
Release : 1994-11-29
Category : Science
ISBN : 9789814502382

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Gene Therapy - From Laboratory To The Clinic by Kam M Hui Pdf

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

Anonim
Nonviral Vectors for Gene Therapy Book PDF

Author : Anonim
Publisher : Academic Press
Page : 296 pages
File Size : 44,9 Mb
Release : 2015-01-23
Category : Science
ISBN : 9780128024706

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Nonviral Vectors for Gene Therapy by Anonim Pdf

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

Translating MicroRNAs to the Clinic

Jeffrey Laurence
Translating MicroRNAs to the Clinic Book PDF

Author : Jeffrey Laurence
Publisher : Academic Press
Page : 392 pages
File Size : 52,5 Mb
Release : 2016-09-17
Category : Science
ISBN : 9780128005545

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Translating MicroRNAs to the Clinic by Jeffrey Laurence Pdf

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

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

Jonathan Kimmelman
Gene Transfer and the Ethics of First in Human Research Book PDF

Author : Jonathan Kimmelman
Publisher : Cambridge University Press
Page : 219 pages
File Size : 48,7 Mb
Release : 2010
Category : Law
ISBN : 9780521690843

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Gene Transfer and the Ethics of First-in-Human Research by Jonathan Kimmelman Pdf

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

Gene Therapy for Cancer

Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Gene Therapy for Cancer Book PDF

Author : Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Publisher : Springer Science & Business Media
Page : 469 pages
File Size : 47,5 Mb
Release : 2007-10-26
Category : Medical
ISBN : 9781597452229

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Gene Therapy for Cancer by Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher Pdf

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.

Gene Therapy Technologies, Applications and Regulations

Anthony Meager
Gene Therapy Technologies Applications and Regulations Book PDF

Author : Anthony Meager
Publisher : John Wiley & Sons
Page : 448 pages
File Size : 54,8 Mb
Release : 1999-11-05
Category : Medical
ISBN : UOM:39015047870202

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Gene Therapy Technologies, Applications and Regulations by Anthony Meager Pdf

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.

From Research to Reality

The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada
From Research to Reality Book PDF

Author : The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada
Publisher : Council of Canadian Academies
Page : 122 pages
File Size : 48,5 Mb
Release : 2020-11-05
Category : Medical
ISBN : 9781926522791

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From Research to Reality by The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada Pdf

From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them.

Pharmaceutical Biotechnology

Daan J. A. Crommelin,Robert D. Sindelar
Pharmaceutical Biotechnology Book PDF

Author : Daan J. A. Crommelin,Robert D. Sindelar
Publisher : CRC Press
Page : 456 pages
File Size : 40,7 Mb
Release : 2002-11-14
Category : Medical
ISBN : 0415285011

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Pharmaceutical Biotechnology by Daan J. A. Crommelin,Robert D. Sindelar Pdf

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.

Advancing Gene-Targeted Therapies for Central Nervous System Disorders

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Advancing Gene Targeted Therapies for Central Nervous System Disorders Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
Page : 93 pages
File Size : 49,6 Mb
Release : 2019-12-01
Category : Medical
ISBN : 9780309495844

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Advancing Gene-Targeted Therapies for Central Nervous System Disorders by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders Pdf

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.

Molecular and Cellular Therapies for Motor Neuron Diseases

Nicholas M Boulis,Deirdre O’Connor,Anthony Donsante
Molecular and Cellular Therapies for Motor Neuron Diseases Book PDF

Author : Nicholas M Boulis,Deirdre O’Connor,Anthony Donsante
Publisher : Academic Press
Page : 336 pages
File Size : 44,8 Mb
Release : 2017-01-18
Category : Psychology
ISBN : 9780128025246

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Molecular and Cellular Therapies for Motor Neuron Diseases by Nicholas M Boulis,Deirdre O’Connor,Anthony Donsante Pdf

Molecular and Cellular Therapies for Motor Neuron Diseases discusses the basics of the diseases, also covering advances in research and clinical trials. The book provides a resource for students that will help them learn the basics in a detailed manner that is required for scientists and clinicians. Users will find a comprehensive overview of the background of Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig’s Disease) and Spinal Muscular Atrophy (SMA), along with the current understanding of their genetics and mechanisms. In addition, the book details gene and cell therapies that have been developed and their translation to clinical trials. Provides an overview of gene and cell therapies for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases Edited by a leading Neurosurgeon and two research scientists to promote synthesis between basic neuroscience and clinical relevance Presents a great resource for researchers and practitioners in neuroscience, neurology, and gene and cell therapy

A Guide to Human Gene Therapy

Roland W. Herzog,Sergei Zolotukhin
A Guide to Human Gene Therapy Book PDF

Author : Roland W. Herzog,Sergei Zolotukhin
Publisher : World Scientific
Page : 415 pages
File Size : 42,9 Mb
Release : 2010
Category : Medical
ISBN : 9789814280914

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A Guide to Human Gene Therapy by Roland W. Herzog,Sergei Zolotukhin Pdf

1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne

Enabling America

Institute of Medicine,Committee on Assessing Rehabilitation Science and Engineering
Enabling America Book PDF

Author : Institute of Medicine,Committee on Assessing Rehabilitation Science and Engineering
Publisher : National Academies Press
Page : 423 pages
File Size : 54,8 Mb
Release : 1997-11-24
Category : Medical
ISBN : 9780309063746

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Enabling America by Institute of Medicine,Committee on Assessing Rehabilitation Science and Engineering Pdf

The most recent high-profile advocate for Americans with disabilities, actor Christopher Reeve, has highlighted for the public the economic and social costs of disability and the importance of rehabilitation. Enabling America is a major analysis of the field of rehabilitation science and engineering. The book explains how to achieve recognition for this evolving field of study, how to set priorities, and how to improve the organization and administration of the numerous federal research programs in this area. The committee introduces the "enabling-disability process" model, which enhances the concepts of disability and rehabilitation, and reviews what is known and what research priorities are emerging in the areas of: Pathology and impairment, including differences between children and adults. Functional limitationsâ€"in a person's ability to eat or walk, for example. Disability as the interaction between a person's pathologies, impairments, and functional limitations and the surrounding physical and social environments. This landmark volume will be of special interest to anyone involved in rehabilitation science and engineering: federal policymakers, rehabilitation practitioners and administrators, researchers, and advocates for persons with disabilities.

Gene Therapy

Mauro Giacca
Gene Therapy Book PDF

Author : Mauro Giacca
Publisher : Springer Science & Business Media
Page : 318 pages
File Size : 51,9 Mb
Release : 2010-11-01
Category : Medical
ISBN : 9788847016439

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Gene Therapy by Mauro Giacca Pdf

I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided.