Translating Gene Therapy to the Clinic Book

Translating Gene Therapy to the Clinic


  • Author : Jeffrey Laurence
  • Publisher : Academic Press
  • Release Date : 2014-11-14
  • Genre: Science
  • Pages : 346
  • ISBN 10 : 9780128005644

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Translating Gene Therapy to the Clinic Excerpt :

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 Book

Translating Regenerative Medicine to the Clinic


  • Author : Jeffrey Laurence
  • Publisher : Academic Press
  • Release Date : 2015-11-18
  • Genre: Science
  • Pages : 354
  • ISBN 10 : 9780128005521

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Translating Regenerative Medicine to the Clinic Excerpt :

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

Translating MicroRNAs to the Clinic Book

Translating MicroRNAs to the Clinic


  • Author : Jeffrey Laurence
  • Publisher : Academic Press
  • Release Date : 2016-09-17
  • Genre: Science
  • Pages : 392
  • ISBN 10 : 9780128005545

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Translating MicroRNAs to the Clinic Excerpt :

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 Therapy Book

Gene Therapy


  • Author : Kam M. Hui
  • Publisher : World Scientific
  • Release Date : 1994
  • Genre: Medical
  • Pages : 278
  • ISBN 10 : 9810216556

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Gene Therapy Excerpt :

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.

Advancing Gene Targeted Therapies for Central Nervous System Disorders Book

Advancing Gene Targeted Therapies for Central Nervous System Disorders


  • Author : National Academies of Sciences, Engineering, and Medicine
  • Publisher : National Academies Press
  • Release Date : 2019-10-31
  • Genre: Medical
  • Pages : 92
  • ISBN 10 : 9780309495875

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Advancing Gene Targeted Therapies for Central Nervous System Disorders Excerpt :

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 Transfer and the Ethics of First in Human Research Book

Gene Transfer and the Ethics of First in Human Research


  • Author : Jonathan Kimmelman
  • Publisher : Cambridge University Press
  • Release Date : 2009-10-29
  • Genre: Law
  • Pages : 205
  • ISBN 10 : 9780521690843

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Gene Transfer and the Ethics of First in Human Research Excerpt :

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

Epidemiology of Endocrine Tumors Book

Epidemiology of Endocrine Tumors


  • Author : Jahangir Moini
  • Publisher : Elsevier
  • Release Date : 2021-02-17
  • Genre: Medical
  • Pages : 568
  • ISBN 10 : 9780128232668

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Epidemiology of Endocrine Tumors Excerpt :

Epidemiology of Endocrine Tumors brings current data and clinical research into one source for a multidisciplinary audience. The book discusses the prevalence, incidence, etiology, pathology, diagnosis and treatment of various endocrine tumors. With clear and focused writing, it is essential reading for healthcare professionals, endocrinologists, oncologists, and public health professionals. Users will be able to bridge the knowledge gap that exists in the comprehensive coverage surrounding the epidemiology of endocrine tumors. Globally, the prevalence and incidence of endocrine tumors is high. This audience needs a treatise where they can gain a broad overview of endocrine tumors with a focus on epidemiology. Supplies information about the epidemiology of various endocrine tumors, both benign and malignant, to endocrinologists, oncologists and related health care professionals Focuses on the impact upon costs and patient deaths due to complications of these tumors Describes how endocrine tumors affect various age groups and ethnicities, discussing the prevention of endocrine tumors Presents chapters on Cancer Problem, Specific Endocrine Tumors, Prevention, Detection and Diagnosis, and Treatment of Endocrine Tumors Provides review questions with an answer key and detailed glossary

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

Gene Therapy of the Central Nervous System From Bench to Bedside


  • Author : Michael G. Kaplitt
  • Publisher : Gulf Professional Publishing
  • Release Date : 2006
  • Genre: Medical
  • Pages : 355
  • ISBN 10 : 0123976324

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

Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far 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 of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and 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 of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use

Guide to Cell Therapy GxP Book

Guide to Cell Therapy GxP


  • Author : Joaquim Vives
  • Publisher : Academic Press
  • Release Date : 2015-07-24
  • Genre: Medical
  • Pages : 266
  • ISBN 10 : 9780128031162

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Guide to Cell Therapy GxP Excerpt :

Guide to Cell Therapy GxP is a practical guide to the implementation of quality assurance systems for the successful performance of all cell-based clinical trials. The book covers all information that needs to be included in investigational medicinal product dossier (IMPD), the launching point for any clinical investigation, and beyond. Guide to Cell Therapy GxP bridges a knowledge gap with the inclusion of examples of design of GLP-compliant preclinical studies; design of bioprocesses for autologous/allogeneic therapies; and instruction on how to implement GLP/GMP standards in centers accredited with other quality assurance standards. Guide to Cell Therapy GxP is an essential resource for scientists and researchers in hospitals, transfusion centers, tissue banks, and other research institutes who may not be familiar with the good scientific practice regulations that were originally designed for product development in corporate environments. This book is also a thorough resource for PhD students, Post-docs, Principal Investigators, Quality Assurance Units, and Government Inspectors who want to learn more about how quality standards are implemented in public institutions developing cell-based products. Easy access to important information on current regulations, state-of-the-art techniques, and recent advances otherwise scattered on various funding websites, within conference proceedings, or maintained in local knowledge Features protocols, techniques for trouble-shooting common problems, and an explanation of the advantages and limitations of a technique in generating conclusive data Includes practical examples of successful implementation of quality standards

Molecular Biology of Cancer  Translation to the Clinic Book

Molecular Biology of Cancer Translation to the Clinic


  • Author : Anonim
  • Publisher : Academic Press
  • Release Date : 2010-12-16
  • Genre: Science
  • Pages : 400
  • ISBN 10 : 012385072X

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Molecular Biology of Cancer Translation to the Clinic Excerpt :

Advances in molecular biology over the last several decades are being steadily applied to our understanding of the molecular biology of cancer, and these advances in knowledge are being translated into the clinical practice of oncology. This volume explores some of the most exciting recent advances in basic research on the molecular biology of cancer and how this knowledge is leading to advances in the diagnosis, treatment, and prevention of cancer. * This series provides a forum for discussion of new discoveries, approaches, and ideas * Contributions from leading scholars and industry experts * Reference guide for researchers involved in molecular biology and related fields

Gene Delivery Book

Gene Delivery


  • Author : Yashwant Pathak
  • Publisher : CRC Press
  • Release Date : 2022-06-29
  • Genre: Medical
  • Pages : 246
  • ISBN 10 : 9781000594799

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Gene Delivery Excerpt :

Gene delivery is a transport of genes of therapeutic values into the chromosomes of the cells or tissues which can be targeted to replace the faulty genes. In last two decades lot of research efforts are dedicated to gene delivery for therapeutic applications. Today gene therapy is promising approach in treatment of genetic diseases including mitochondrial related diseases like blindness, muscular dystrophy, cystic fibrosis, and some cancers. Gene Delivery Systems: Nano Delivery Technologies observes the exploration of nanotechnology for gene therapy and gene delivery. Written by prominent authors in the field, this book covers various aspects of gene delivery including challenges in delivering gene therapy, advances in genome editing, RNA-based gene therapy, Green nanoparticles for oligonucleotide delivery. Additional features include" Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing. Includes knowledge of the current application of CRISPR/Cas9 gene-editing technique; an approach that has recently been given the Noble Prize. Examines the development of mRNA vaccines for Covid -19 in challenging pandemic scenario Discusses siRNA, mRNA, and DNA plasmids.

Translational Neuroscience Book

Translational Neuroscience


  • Author : Mark H. Tuszynski
  • Publisher : Springer
  • Release Date : 2016-03-08
  • Genre: Medical
  • Pages : 584
  • ISBN 10 : 9781489976543

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Translational Neuroscience Excerpt :

Translational Neuroscience offers a far-reaching and insightful series of perspectives on the effort to bring potentially revolutionary new classes of therapies to the clinic, thereby transforming the treatment of human nervous system disorders. Great advances in the fields of basic neuroscience, molecular biology, genomics, gene therapy, cell therapy, stem cell biology, information technology, neuro devices, rehabilitation and others over the last 20 years have generated unprecedented opportunities to treat heretofore untreatable disorders of the nervous system. This book provides a wide-ranging yet detailed sample of many of these efforts, together with the methods for pursuing clinical translation and assessing clinical outcomes. Among the topics covered are Alzheimer’s disease, Parkinson’s disease, stroke, multiple sclerosis, epilepsy, motor neuron disease, pain, inborn errors of metabolism, brain tumors, spinal cord injury, neuroprosthetics, rehabilitation and clinical trial design/consideration. Translational Neuroscience is aimed at basic neuroscientists, translational neuroscientists and clinicians who seek to gain a perspective on the nature and promise of translational therapies in the current era. Both students and established professionals will benefit from the content.

Placental Fetal Growth Restriction Book

Placental Fetal Growth Restriction


  • Author : Christoph Lees
  • Publisher : Cambridge University Press
  • Release Date : 2018-08-02
  • Genre: Medical
  • Pages : 294
  • ISBN 10 : 9781107101395

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Placental Fetal Growth Restriction Excerpt :

Master the effective evaluation of placental-fetal growth restriction (PFGR), whilst reducing the risk of perinatal mortality and morbidity in patients worldwide.

Animal Models of Molecular Pathology Book

Animal Models of Molecular Pathology


  • Author : P. Michael Conn
  • Publisher : Academic Press
  • Release Date : 2011-11-26
  • Genre: Science
  • Pages : 472
  • ISBN 10 : 9780123948311

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Animal Models of Molecular Pathology Excerpt :

This volume explores some of the most exciting recent advances in basic research on animal models of molecular pathology. This series provides a forum for discussion of new discoveries, approaches, and ideas Contributions from leading scholars and industry experts Reference guide for researchers involved in molecular biology and related fields

1st World Congress on Electroporation and Pulsed Electric Fields in Biology  Medicine and Food   Environmental Technologies Book

1st World Congress on Electroporation and Pulsed Electric Fields in Biology Medicine and Food Environmental Technologies


  • Author : Tomaz Jarm
  • Publisher : Springer
  • Release Date : 2015-08-31
  • Genre: Technology & Engineering
  • Pages : 455
  • ISBN 10 : 9789812878175

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1st World Congress on Electroporation and Pulsed Electric Fields in Biology Medicine and Food Environmental Technologies Excerpt :

This volume presents the proceedings of the 1st World Congress on Electroporation and Pulsed Electric Fields in Biology, Medicine and Food & Environmental Technologies (WC2015). The congress took place in Portorož, Slovenia, during the week of September 6th to 10th, 2015. The scientific part of the Congress covered different aspects of electroporation and related technologies and included the following main topics: · Application of pulsed electric fields technology in food: challenges and opportunities · Electrical impedance measurement for assessment of electroporation yield · Electrochemistry and electroporation · Electroporation meets electrostimulation · Electrotechnologies for food and biomass treatment · Food and biotechnology applications · In vitro electroporation - basic mechanisms · Interfacial behaviour of lipid-assemblies, membranes and cells in electric fields · Irreversible electroporation in clinical use · Medical applications: electrochemotherapy · Medical applications: gene therapy · Non-electric field-based physical methods inducing cell poration and enhanced molecule transfer · Non-thermal plasmas for food safety, environmental applications and medical treatments · PEF for the food industry: fundamentals and applications · PEF proce ss integration - complex process chains and process combinations in the food industry · Predictable animal models · Pulsed electric fields and electroporation technologies in bioeconomy · Veterinary medical applications