Development Of Gene Therapies

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Exploring Novel Clinical Trial Designs for Gene-Based Therapies

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine
Exploring Novel Clinical Trial Designs for Gene Based Therapies Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine
Publisher : National Academies Press
Page : 127 pages
File Size : 44,8 Mb
Release : 2020-08-27
Category : Medical
ISBN : 9780309672986

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Exploring Novel Clinical Trial Designs for Gene-Based Therapies by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine Pdf

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.

Development of Gene Therapies

Anonim
Development of Gene Therapies Book PDF

Author : Anonim
Publisher : Unknown
Page : 0 pages
File Size : 49,6 Mb
Release : 2024-03
Category : Mathematics
ISBN : 1003230342

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Development of Gene Therapies by Anonim Pdf

"Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community"--

Gene Therapy of Cancer

Stanton L. Gerson,Edmund C. Lattime
Gene Therapy of Cancer Book PDF

Author : Stanton L. Gerson,Edmund C. Lattime
Publisher : Elsevier
Page : 555 pages
File Size : 51,9 Mb
Release : 2002-04-04
Category : Science
ISBN : 9780080491363

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Gene Therapy of Cancer by Stanton L. Gerson,Edmund C. Lattime Pdf

The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Provides in-depth description of targeted systems and treatment strategies Explains the underlying cancer biology necessary for understanding a given therapeutic approach Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses Presents translational focus with emphasis on requirements for clinical implementation Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

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 : 42,6 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.

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 : 51,9 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.

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 : 40,6 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

L. E. Carmichael
Gene Therapy Book PDF

Author : L. E. Carmichael
Publisher : ABDO Publishing Company
Page : 114 pages
File Size : 43,6 Mb
Release : 2013-09-01
Category : Juvenile Nonfiction
ISBN : 9781624013737

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Gene Therapy by L. E. Carmichael Pdf

Amazing medical breakthroughs are made every day. In the past decades, medical researchers have cured diseases that were once deadly and devised new methods to heal that were once unimaginable. This title follows the development of gene therapy, including the discovery of DNA, groundbreaking discoveries and the doctors who made them, and where the science is heading in the future. Learn how gene therapy works and why future applications of the technology will be controversial. Sidebars, full-color photos, a glossary, and well-placed graphs, charts, and maps, enhance this engaging title. Aligned to Common Core Standards and correlated to state standards. Essential Library is an imprint of Abdo Publishing, a division of ABDO.

The Development of Human Gene Therapy

Theodore Friedmann
The Development of Human Gene Therapy Book PDF

Author : Theodore Friedmann
Publisher : CSHL Press
Page : 748 pages
File Size : 48,5 Mb
Release : 1999
Category : Adenoviruses
ISBN : 0879695285

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The Development of Human Gene Therapy by Theodore Friedmann Pdf

The idea of human gene therapy was accepted by the medical community and society at large long before believable clinical benefits began to emerge. In this book, some of the fields most distinguished contributors chronicle the evolution of this momentous direction for medicine, illustrating how imaginative concepts shaped the development of technologies and brought the daring new idea to its current position of imminent practical success. This is a book designed to endure as clinical advances accumulate, a clear-eyed work of reference that will anchor the further development of this revolution in therapy. It is an essential addition to libraries of clinical medicine, biotechnology, and public policy, and a resource that no laboratory investigator with an interest in the biology of gene transfer should be without.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Maria Cristina Galli
Regulatory Aspects of Gene Therapy and Cell Therapy Products Book PDF

Author : Maria Cristina Galli
Publisher : Springer Nature
Page : 243 pages
File Size : 49,7 Mb
Release : 2023-08-01
Category : Science
ISBN : 9783031345678

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Regulatory Aspects of Gene Therapy and Cell Therapy Products by Maria Cristina Galli Pdf

This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Somatic Gene Therapy

Patricia L. Chang
Somatic Gene Therapy Book PDF

Author : Patricia L. Chang
Publisher : CRC Press
Page : 318 pages
File Size : 54,8 Mb
Release : 1994-12-27
Category : Science
ISBN : 0849324408

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Somatic Gene Therapy by Patricia L. Chang Pdf

As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.

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 : 45,5 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.

Cellular Therapy Of Cancer: Development Of Gene Therapy Based Approaches

Robert E Hawkins
Cellular Therapy Of Cancer Development Of Gene Therapy Based Approaches Book PDF

Author : Robert E Hawkins
Publisher : World Scientific
Page : 212 pages
File Size : 55,5 Mb
Release : 2014-05-05
Category : Medical
ISBN : 9789814618533

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Cellular Therapy Of Cancer: Development Of Gene Therapy Based Approaches by Robert E Hawkins Pdf

Cancer research has progressed enormously in recent years. This review volume will address recent findings in the area of T-cell therapy for cancer, including use of tumour infiltrating lymphocytes (TILs) as a therapy for melanoma, choice of target antigens, advances in engineered receptors, methods of gene transfer to T cells, review of cell processing methods and clinical trial design. Written by leadings scientists in the field, this up-to-date review on cancer research will be an important reference source to the researchers and healthcare professionals in the field.

Genome Editing

Toni Cathomen,Matthew Hirsch,Matthew Porteus
Genome Editing Book PDF

Author : Toni Cathomen,Matthew Hirsch,Matthew Porteus
Publisher : Springer
Page : 263 pages
File Size : 44,5 Mb
Release : 2016-03-04
Category : Medical
ISBN : 9781493935093

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Genome Editing by Toni Cathomen,Matthew Hirsch,Matthew Porteus Pdf

This comprehensive volume explores human genetic engineering its pre-clinical and clinical applications, current developments, and as treatment for hereditary diseases. It presents and evaluates the most recent advances in the understanding of mammalian host DNA repair mechanisms, such as double-strand break induced gene targeting and mutagenesis, the development of zinc-finger nucleases, genome editing for neuromuscular diseases, phase integrases, triplex forming oligonucleotides and peptide nucleic acids, aptamer-guided gene targeting, AAV gene editing via DSB repair, engineered nucleases and trinucleotide repeat diseases, and creation of HIV-resistant cells. The expertly authored chapters contextualize current developments within the history of genome editing while also discussing the current and potential safety concerns of this rapidly growing field. Genome Editing: The Next Step in Gene Therapy, the latest volume in the American Society of Gene and Cell Therapy series, deftly illuminates the potential of genetic engineering technology to eradicate today’s deadliest and most prolific diseases. It is ideal reading for clinicians and researchers in genetics and immunology.

Gene Therapy of the Central Nervous System: From Bench to Bedside

Michael G. Kaplitt,Matthew During
Gene Therapy of the Central Nervous System From Bench to Bedside Book PDF

Author : Michael G. Kaplitt,Matthew During
Publisher : Gulf Professional Publishing
Page : 380 pages
File Size : 43,9 Mb
Release : 2006
Category : Medical
ISBN : 0123976324

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Gene Therapy of the Central Nervous System: From Bench to Bedside by Michael G. Kaplitt,Matthew During Pdf

Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system.

An Introduction to Molecular Medicine and Gene Therapy

Thomas F. Kresina
An Introduction to Molecular Medicine and Gene Therapy Book PDF

Author : Thomas F. Kresina
Publisher : John Wiley & Sons
Page : 408 pages
File Size : 48,9 Mb
Release : 2004-03-24
Category : Science
ISBN : 9780471461043

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An Introduction to Molecular Medicine and Gene Therapy by Thomas F. Kresina Pdf

An Introduction to Molecular Medicine and Gene Therapy Edited by Thomas F. Kresina, Ph.D. Gene therapy, or the use of genetic manipulation for disease treatment, is derived from advances in genetics, molecular biology, clinical medicine, and human genomics. Molecular medicine, the application of molecular biological techniques to disease treatment and diagnosis, is derived from the development of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular Medicine and Gene Therapy provides a basis for interpreting new clinical and basic research findings in the areas of cloning, gene transfer, and targeting; the applications of genetic medicine to clinical conditions; ethics and governmental regulations; and the burgeoning fields of genomics, biotechnology, and bioinformatics. By dividing the material into three sections - an introduction to basic science, a review of clinical applications, and a discussion of the evolving issues related to gene therapy and molecular medicine-this comprehensive manual describes the basic approaches to the broad range of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine and Gene Therapy: * Covers new frontiers in gene therapy, animal models, vectors, gene targeting, and ethical/legal considerations * Provides organ-based reviews of current studies in gene therapy for monogenetic, multifactoral or polygenic disorders, and infectious diseases * Includes bold-faced terms, key concepts, summaries, and lists of helpful references by subject in each chapter * Contains appendices on commercial implications and a review of the history of gene therapy This textbook offers a clear, concise writing style, drawing upon the expertise of the authors, all renowned researchers in their respective specialties of molecular medicine. Researchers in genetics and molecular medicine will all find An Introduction to Molecular Medicine and Gene Therapy to be an essential guide to the rapidly evolving field of gene therapy and its applications in molecular medicine.