Genome Based Therapeutics

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Genome-Based Therapeutics

Institute of Medicine,Board on Health Sciences Policy,Roundtable on Translating Genomic-Based Research for Health
Genome Based Therapeutics Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Roundtable on Translating Genomic-Based Research for Health
Publisher : National Academies Press
Page : 89 pages
File Size : 44,5 Mb
Release : 2012-12-21
Category : Medical
ISBN : 9780309260244

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Genome-Based Therapeutics by Institute of Medicine,Board on Health Sciences Policy,Roundtable on Translating Genomic-Based Research for Health Pdf

The number of new drug approvals has remained reasonably steady for the past 50 years at around 20 to 30 per year, while at the same time the total spending on health-related research and development has tripled since 1990. There are many suspected causes for this trend, including increases in regulatory barriers, the rising costs of scientific inquiry, a decrease in research and development efficiency, the downstream effects of patient expirations on investment, and the lack of production models that have successfully incorporated new technology. Regardless, this trajectory is not economically sustainable for the businesses involved, and, in response, many companies are turning toward collaborative models of drug development, whether with other industrial firms, academia, or government. Introducing greater efficiency and knowledge into these new models and aligning incentives among participants may help to reverse the trends highlighted above, while producing more effective drugs in the process. Genome-Based Therapeutics explains that new technologies have the potential to open up avenues of development and to identify new drug targets to pursue. Specifically, improved validation of gene-disease associations through genomics research has the potential to revolutionize drug production and lower development costs. Genetic information has helped developers by increasing their understanding of the mechanisms of disease as well as individual patients' reactions to their medications. There is a need to identify the success factors for the various models that are being developed, whether they are industry-led, academia-led, or collaborations between the two. Genome-Based Therapeutics summarizes a workshop that was held on March 21, 2012, titled New Paradigms in Drug Discovery: How Genomic Data Are Being Used to Revolutionize the Drug Discovery and Development Process. At this workshop the goal was to examine the general approaches being used to apply successes achieved so far, and the challenges ahead.

Genome-based Therapeutics

Anonim
Genome based Therapeutics Book PDF

Author : Anonim
Publisher : Unknown
Page : 109 pages
File Size : 51,6 Mb
Release : 2012
Category : Drugs
ISBN : 0309260213

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Genome-based Therapeutics by Anonim Pdf

The number of new drug approvals has remained reasonably steady for the past 50 years at around 20 to 30 per year, while at the same time the total spending on health-related research and development has tripled since 1990. There are many suspected causes for this trend, including increases in regulatory barriers, the rising costs of scientific inquiry, a decrease in research and development efficiency, the downstream effects of patient expirations on investment, and the lack of production models that have successfully incorporated new technology. Regardless, this trajectory is not economically sustainable for the businesses involved, and, in response, many companies are turning toward collaborative models of drug development, whether with other industrial firms, academia, or government. Introducing greater efficiency and knowledge into these new models and aligning incentives among participants may help to reverse the trends highlighted above, while producing more effective drugs in the process. Genome-based therapeutics explains that new technologies have the potential to open up avenues of development and to identify new drug targets to pursue. Specifically, improved validation of gene-disease associations through genomics research has the potential to revolutionize drug production and lower development costs. Genetic information has helped developers by increasing their understanding of the mechanisms of disease as well as individual patients' reactions to their medications. There is a need to identify the success factors for the various models that are being developed, whether they are industry-led, academia-led, or collaborations between the two. Genome-Based Therapeutics summarizes a workshop that was held on March 21, 2012, titled New paradigms in drug discovery: how genomic data are being used to revolutionize the drug discovery and development process. At this workshop the goal was to examine the general approaches being used to apply successes achieved so far, and the challenges ahead"--Publisher's description

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 : 54,9 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.

Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests

Institute of Medicine,Board on Health Sciences Policy,Roundtable on Translating Genomic-Based Research for Health
Refining Processes for the Co Development of Genome Based Therapeutics and Companion Diagnostic Tests Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Roundtable on Translating Genomic-Based Research for Health
Publisher : National Academies Press
Page : 102 pages
File Size : 50,6 Mb
Release : 2014-03-06
Category : Medical
ISBN : 9780309298247

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Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests by Institute of Medicine,Board on Health Sciences Policy,Roundtable on Translating Genomic-Based Research for Health Pdf

Many drug developers have examined new strategies for creating efficiencies in their development processes, including the adoption of genomics-based approaches. Genomic data can identify new drug targets for both common and rare diseases, can predict which patients are likely to respond to a specific treatment, and has the potential to significantly reduce the cost of clinical trials by reducing the number of patients that must be enrolled in order to demonstrate safety and efficacy. A key component of the approval of targeted therapeutics is the ability to identify the population of patients who will benefit from treatment, and this has largely hinged on the co-development and co-submission to the FDA of a companion diagnostic test.The co-development process, or the development of the test and drug for the simultaneous submission to FDA, has led to a major alteration in the way that drugs are being developed, with traditionally separate entities-pharmaceutical and diagnostic companies-now working in close collaboration. Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests is the summary of a workshop held by the Roundtable on Translating Genomic-Based Research for Health on February 27, 2013 to examine and discuss challenges and potential solutions for the codevelopment of targeted therapeutics and companion molecular tests for the prediction of drug response. Prior to the workshop, key stakeholders, including laboratory and medical professional societies, were individually asked to provide possible solutions to resolve the concerns raised about co-development of companion diagnostic tests and therapies. Workshop speakers were charged with addressing these solutions in their presentations by providing insight on (1) whether the proposed solutions address the problems described, (2) whether there are other solutions to propose, and (3) what steps could be taken to effectively implement the proposed solutions.

Fast Facts: Gene Therapy

Roland Herzog,Linda Popplewell
Fast Facts Gene Therapy Book PDF

Author : Roland Herzog,Linda Popplewell
Publisher : Karger Medical and Scientific Publishers
Page : 102 pages
File Size : 46,8 Mb
Release : 2020-04-08
Category : Medical
ISBN : 9783318066661

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Fast Facts: Gene Therapy by Roland Herzog,Linda Popplewell Pdf

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

Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders

Nicola Brunetti-Pierri
Safety and Efficacy of Gene Based Therapeutics for Inherited Disorders Book PDF

Author : Nicola Brunetti-Pierri
Publisher : Springer
Page : 220 pages
File Size : 47,5 Mb
Release : 2017-05-22
Category : Medical
ISBN : 9783319534572

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Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders by Nicola Brunetti-Pierri Pdf

In this book, leading international experts analyze state-of-the-art advances in gene transfer vectors for applications in inherited disorders and also examine the toxicity profiles of these methods. The authors discuss the strengths and weaknesses of available vectors in the clinical setting, and specifically focus on the challenges and possible solutions that researchers are testing in order to improve the safety of gene therapy for genetic diseases. This comprehensive and authoritative overview of vector development is a necessary text for researchers, toxicologists, pharmacologists, molecular biologists, physicians, and students in these fields.

Somatic Gene Therapy

Patricia L. Chang
Somatic Gene Therapy Book PDF

Author : Patricia L. Chang
Publisher : CRC Press
Page : 318 pages
File Size : 48,7 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 : 40,8 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.

Second Generation Cell and Gene-Based Therapies

Alain Vertes,Nathan J. Dowden,Devyn Smith,Nasib Qureshi
Second Generation Cell and Gene Based Therapies Book PDF

Author : Alain Vertes,Nathan J. Dowden,Devyn Smith,Nasib Qureshi
Publisher : Academic Press
Page : 846 pages
File Size : 46,6 Mb
Release : 2020-02-07
Category : Science
ISBN : 9780128120330

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Second Generation Cell and Gene-Based Therapies by Alain Vertes,Nathan J. Dowden,Devyn Smith,Nasib Qureshi Pdf

Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization "Perspectives" section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies

Manufacturing of Gene Therapeutics

G. Subramanian
Manufacturing of Gene Therapeutics Book PDF

Author : G. Subramanian
Publisher : Springer Science & Business Media
Page : 355 pages
File Size : 46,6 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9781461513537

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Manufacturing of Gene Therapeutics by G. Subramanian Pdf

Advances in molecular biology and recombinant DNA technology have accelerated progress in many fields of life science research, including gene therapy. A large number of genetic engineering approaches and methods are readily available for gene cloning and therapeutic vector construction. Significant progress is being made in genomic, DNA sequencing, gene expression, gene delivery and cloning. Thus gene therapy has already shown that it holds great promise for the treatment of many diseases and disorders. In general it involves the delivery of recombinant genes or transgenes into somatic cells to replace proteins with a genetic defect or to transfer with the pathological process of an illness. The viral and non-viral delivery systems may hold the potential for future non-invasive, cost-effective oral therapy of genetically-based disorders. Recent years have seen considerable progress in the discovery and early clinical development of a variety of gene therapeutic products. The availability, validation, and implementation of gene therapeutic products has also enabled success in testing and evaluation. New challenges will need to be overcome to ensure that products will also be successful in later clinical development and ultimately for marketing authorisation. These new challenges will include improvements in delivery systems, better control of in-vivo targeting, increased level transduction and duration of expression of the gene, and manufacturing process efficiencies that enable reduction in production costs. Perhaps profound understanding of regulated gene design may result in innovative bioproducts exhibiting safety and efficacy profiles that are significantly superior to those achieved by the use of naturally occurring genes. This procedure may contribute considerably to fulfilling standards set by regulatory authorities. This book provides an overview of the current advances in the field of gene therapy and the methods that are being successfully applied in the manufacture of gene therapeutic products, and hopefully will stimulate further progress and advancement in this field to meet the ever-increasing demands.

Gene and Cell Therapy

Nancy Smyth Templeton
Gene and Cell Therapy Book PDF

Author : Nancy Smyth Templeton
Publisher : CRC Press
Page : 894 pages
File Size : 55,5 Mb
Release : 2003-12-17
Category : Science
ISBN : 9780824758608

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Gene and Cell Therapy by Nancy Smyth Templeton Pdf

This reference is completely revised and expanded to reflect the most critical studies, controversies, and technologies impacting the medical field, including probing research on lentivirus, gutless adenovirus, bacterial and baculovirus vectors, retargeted viral vectors, in vivo electroporation, in vitro and in vivo gene detection systems, and all inducible gene expression systems. Scrutinizing every tool, technology, and issue impacting the future of gene and cell research, it is specifically written and organized for laymen, scholars, and specialists from varying backgrounds and disciplines to understand the current status of gene and cell therapy and anticipate future developments in the field.

Gene Therapy

Nancy Smyth Templeton,D. D. Lasic
Gene Therapy Book PDF

Author : Nancy Smyth Templeton,D. D. Lasic
Publisher : CRC Press
Page : 616 pages
File Size : 51,9 Mb
Release : 2000-06-15
Category : Medical
ISBN : UOM:39015050321036

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Gene Therapy by Nancy Smyth Templeton,D. D. Lasic Pdf

This text covers all aspects of gene therapy, including basic principles, viral and nonviral delivery systems, targeted diseases, regulatory issues, and FDA requirements. It investigates genetic bullets to block HIV-1 replication and genetic guns to deliver antiviral agents.

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 : 51,8 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.

Gene Therapeutics

Jon A. Wolff
Gene Therapeutics Book PDF

Author : Jon A. Wolff
Publisher : Springer Science & Business Media
Page : 431 pages
File Size : 41,6 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9781468468229

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Gene Therapeutics by Jon A. Wolff Pdf

During the first half century of genetics, coinciding with the first half of this cen tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex tremely efficient in transferring their contents to the chromosomes of cultured cells.

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 : 47,9 Mb
Release : 2020-07-27
Category : Medical
ISBN : 9780309673013

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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.