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Translating Gene Therapy To The Clinic

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Translating Gene Therapy to the Clinic

Translating Gene Therapy to the Clinic Book
Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Release : 2014-11-14
ISBN : 0128005645
Language : En, Es, Fr & De

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Book Description :

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

Translating Regenerative Medicine to the Clinic Book
Author : Jeffrey Laurence,Pedro Baptista,Anthony Atala
Publisher : Academic Press
Release : 2015-11-18
ISBN : 0128005521
Language : En, Es, Fr & De

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Book Description :

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

Gene Therapy Book
Author : Kam M. Hui
Publisher : World Scientific
Release : 1994
ISBN : 9789810216559
Language : En, Es, Fr & De

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Book Description :

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.

Translating Epigenetics to the Clinic

Translating Epigenetics to the Clinic Book
Author : Jeffrey Laurence
Publisher : Academic Press
Release : 2016-12-30
ISBN : 0128006129
Language : En, Es, Fr & De

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Book Description :

Translating Epigenetics to the Clinic reviews current methodological tools and experimental approaches used by leading translational researchers seeking to use epigenetics as a clinical model. It organizes epigenetics into disease treatment areas with a major focus on oncology, and with much coverage of pervasive treatment categories such as diabetes, as well as the ‘diseases of modernity’—including pharmacological addiction, dementia, and ageing. Pedagogically, the work concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field. The book promotes cross-disciplinary communication between the sub-specialties of medicine. In common with the rest of the books in Translational Medicine, the book remains unified in theme by emphasizing recent innovations, critical barriers to progress, and the new tools being used to overcome them. Also includes 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 epigenetics in the lab and clinic Features extensive pedagogical updates aiming to improve the education of translational researchers in this field Offers a transdisciplinary approach to support cross-fertilization between different sub-specialties of medicine

Concepts in Genetic Medicine

Concepts in Genetic Medicine Book
Author : Boro Dropulic,Barrie Carter
Publisher : John Wiley & Sons
Release : 2008-01-03
ISBN : 9780470184578
Language : En, Es, Fr & De

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Book Description :

Download Concepts in Genetic Medicine book written by Boro Dropulic,Barrie Carter, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

The Clinibook

The Clinibook Book
Author : Odile Cohen-Haguenauer
Publisher : EDK Editions
Release : 2013-05-03T00:00:00+02:00
ISBN : 2842542371
Language : En, Es, Fr & De

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Book Description :

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

Recent Advances in the Biology Therapy and Management of Melanoma

Recent Advances in the Biology  Therapy and Management of Melanoma Book
Author : Lester Davids
Publisher : IntechOpen
Release : 2013-02-13
ISBN : 9789535109761
Language : En, Es, Fr & De

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Book Description :

The book Recent Advances in the Biology, Therapy and Management of Melanoma brings up-to-date information regarding a number of aspects which culminate in illuminating potential targets in the fight against melanoma. This book is intended to be a reference book for both the scientific and clinical communities and brings complicated subject matter together in an easy, readable way. Undoubtedly fundamental scientific understanding has to then be translated to the clinic in order for us to make significant strides in eradicating melanoma. It is hoped that scientists, clinicians, students and residents find this book useful in their studies on melanoma and that it not only expands their perspectives and views on the field, but challenges them to forge ahead towards discovering the ultimate cure.

Molecular Biology of Cancer Translation to the Clinic

Molecular Biology of Cancer  Translation to the Clinic Book
Author : Anonim
Publisher : Academic Press
Release : 2010-12-16
ISBN : 9780123850720
Language : En, Es, Fr & De

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Book Description :

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

Placental Fetal Growth Restriction

Placental Fetal Growth Restriction Book
Author : Christoph Lees,Gerard H. A. Visser,Kurt Hecher
Publisher : Cambridge University Press
Release : 2018-08-02
ISBN : 1107101395
Language : En, Es, Fr & De

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Book Description :

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

Epidemiology of Endocrine Tumors

Epidemiology of Endocrine Tumors Book
Author : Jahangir Moini,Craig Badolato,Raheleh Ahangari
Publisher : Elsevier
Release : 2021-02-17
ISBN : 0128232668
Language : En, Es, Fr & De

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Book Description :

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

Translating MicroRNAs to the Clinic

Translating MicroRNAs to the Clinic Book
Author : Jeffrey Laurence
Publisher : Academic Press
Release : 2016-09-17
ISBN : 0128005548
Language : En, Es, Fr & De

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Book Description :

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

Translational Neuroscience

Translational Neuroscience Book
Author : Mark H. Tuszynski
Publisher : Springer
Release : 2016-03-08
ISBN : 148997654X
Language : En, Es, Fr & De

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Book Description :

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.

Guide to Cell Therapy GxP

Guide to Cell Therapy GxP Book
Author : Joaquim Vives,Gloria Carmona
Publisher : Academic Press
Release : 2015-07-24
ISBN : 0128031166
Language : En, Es, Fr & De

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Book Description :

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

Animal Models of Molecular Pathology

Animal Models of Molecular Pathology Book
Author : P. Michael Conn
Publisher : Academic Press
Release : 2012
ISBN : 0123945968
Language : En, Es, Fr & De

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Book Description :

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

1st World Congress on Electroporation and Pulsed Electric Fields in Biology  Medicine and Food   Environmental Technologies Book
Author : Tomaz Jarm,Peter Kramar
Publisher : Springer
Release : 2015-08-31
ISBN : 9812878173
Language : En, Es, Fr & De

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Book Description :

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

Nanoparticle Cloaking of Viral Vectors for Enhanced Gene Delivery

Nanoparticle Cloaking of Viral Vectors for Enhanced Gene Delivery Book
Author : Ajay Ajit Sapre
Publisher : Unknown
Release : 2018
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Gene therapy has the potential to treat a wide range of diseases and ailments from cancer to blindness by altering or overcoming disease at its genetic roots. This is accomplished by adding or alternating genetic information of diseased tissue or at a distant site for systemic treatment. Genetically modified viruses are the most efficient tools for delivery of genes, but have significant side effects that have limited the success of clinical trials. Adenovirus (Ad) is a DNA virus that has been tested in over 100 clinical trials and is the focus of this dissertation. Innate and adaptive immune responses, hepatic clearance, and cellular tropism are the primary causes of poor Ad clinical translation. Cloaking technologies using synthetic or biologic materials have the potential to overcome these issues. Chapter 2 & 3 describes a method to address clinical barriers by encapsulating Ad in silica as a nanoparticle formulation. Silica is biodegradeable, biocompatible, and used in variety of nanoparticle formulations to enhance drug delivery. Silica encapsulated Ad (SiAd) enhances transduction and expands tropism in vitro. In immune-compromised mice, SiAd enhanced tumor transduction while reducing liver uptake and in immune-competent mice, SiAd reduced both the innate and adaptive immune response against Ad. As a model for cancer gene therapy, we used Ad expressing TNF-related apoptosis-inducing ligand (TRAIL) and show inhibited tumor growth with SiAd-TRAIL. In chapter 4, we explore applying the concepts of the previous chapters to Adeno-associated virus (AAV), which is another viral vector gaining traction for gene therapy in the clinic and one that is subject to similar barriers to clinical success as Ad. We explore exosome membrane cloaking and silica cloaking as methods to enhance AAV transduction in vitro. Overall, this dissertation covers techniques that seek to merge the efficiency of viral gene expression with the versatility of nanoparticle technology to address clinical challenges in the field of gene therapy.

Fetal Stem Cells in Regenerative Medicine

Fetal Stem Cells in Regenerative Medicine Book
Author : Dario O. Fauza,Mahmud Bani
Publisher : Springer
Release : 2016-05-02
ISBN : 149393483X
Language : En, Es, Fr & De

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Book Description :

This book explores the regenerative properties of fetal stem cells, from feto-maternal cell traffic through perinatal stem cells, with a discussion of key topics including stem cell banking, drug screening, in utero stem cell transplantation and ethical considerations. The expertly authored chapters also delve into embryonic, amniotic membrane, and umbilical cord blood stem cells; fetal development models; fetal cell reprogramming; culture methods; disease models; perinatal gene therapy, and more. These chapters are grouped into four sections, each discussing a separate prenatal stem cell population and providing fascinating historical contexts for our knowledge of these systems. Featuring a foreword written by the renowned Dr. Joseph Vacanti of the Harvard Stem Cell Institute, Fetal Stem Cells in Regenerative Medicine: Principles and Translational Strategies is a welcome and timely contribution to the Stem Cell Biology and Regenerative Medicine series. It is essential reading for scientists and researchers, clinicians and residents, and advanced students involved in stem cells, regenerative medicine, tissue engineering, and related disciplines such as embryology.

Gene Therapy From Laboratory to the Clinic

Gene Therapy     From Laboratory to the Clinic Book
Author : Kam M Hui
Publisher : World Scientific
Release : 1994-11-29
ISBN : 9814502383
Language : En, Es, Fr & De

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Book Description :

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. Contents:Foreword (R A Weiss)An Introduction to Gene Therapy and Cancer (Y H Tan)Clinical Strategies and Expectations for the Molecular Biotherapy of Cancer (H K B Silver & R J Klasa)Tumor-Directed Cytokine Gene Therapy (C S Chiang et al.)Therapeutic Applications of Antisense Nuclei Acids (T M C Tan et al.)Molecular Targeting of Cancer: Retroviral Vector-Mediated Antisense Nuclei Acid Therapy (Y Zhang et al.)DC-Chol Liposomes as DNA Carriers for Gene Therapy (A Singhal & L Huang)Accell Particle Bombardment for Gene Transfer as a Useful Gene Therapy Technique (N-S Yang et al.)DNA-Coated Microprojectiles for Gene Delivery into Live Animals (D-C Tang & S A Johnston)Expression of “Foreign” Class I Major Histocompatibility Complex (MHC) Molecules on Experimental Tumors and the Induction of Active Specific Cancer Immunotherapy (K M Hui)Gene Transfer to the Central Nervous System Mediated by Herpes Simplex Virus Vectors (J C Glorioso et al.)Adeno-Associated Virus-Based Vectors for Human Gene Therapy (R J Samulski)Index Readership: Scientists, clinical investigators, physicians and graduate students in medical/ biological sciences. keywords:Human Gene Therapy;Cancer Gene Therapy;DNA Vaccines;Gene Transfer;Viral Vector;Adenovirus;Retrovirus;Non-Viral Vector;Liposomes;Gene Gun “… the format and length of this text makes it an easy to read introduction to the area of gene therapy. It would be ideally suited to the reader with a working knowledge of one particular area of gene therapy who wished to gain a broader perspective of the field. Medical scientists in particular would find this a useful adjunct to their current textbooks.” Cancer Forum

Safety Ethics and Regulations

Safety  Ethics and Regulations Book
Author : Phuc Van Pham,Achim Rosemann
Publisher : Springer
Release : 2017-08-12
ISBN : 3319591657
Language : En, Es, Fr & De

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Book Description :

This invaluable resource discusses the saftey, ethics, and regulations of developing stem cell clinical applications. Each chapter is contributed by a preeminent scientist in the field and covers such topics as clinical safety of stem cell gene therapy, the patentability of hESC technologies, international guidelines, challenges to international stem cell clinical trials, worldwide regulations including in emerging markets like China and Taiwan. Saftey, Ethics, and Regulations and the other books in the Stem Cells in Clinical Applications series will be invaluable to scientists, researchers, advanced students and clinicians working in stem cells, regenerative medicine or tissue engineering.

Gene and Cell Therapies for Beta Globinopathies

Gene and Cell Therapies for Beta Globinopathies Book
Author : Punam Malik,John Tisdale
Publisher : Springer
Release : 2017-11-09
ISBN : 1493972995
Language : En, Es, Fr & De

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Book Description :

Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, which are often available only to 15-20% of patients. An alternative to allogeneic HS CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances.