Safety And Efficacy Of Gene Based Therapeutics For Inherited Disorders
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Safety And Efficacy Of Gene Based Therapeutics For Inherited Disorders
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Author : Nicola Brunetti-Pierri
language : en
Publisher: Springer
Release Date : 2017-05-22
Safety And Efficacy Of Gene Based Therapeutics For Inherited Disorders written by Nicola Brunetti-Pierri and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-05-22 with Medical categories.
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.
Advances In Biotechnology
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Author : Indu Ravi
language : en
Publisher: Springer Science & Business Media
Release Date : 2013-10-21
Advances In Biotechnology written by Indu Ravi and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2013-10-21 with Science categories.
The book “Advances in Biotechnology” is about recent advances in some of the important fields that are ongoing in certain biotechnological applications. Biotechnology has been quite helpful in keeping pace with the demands of every increasing human population and in improving the quality of human life. Major biotechnological achievements associated with human welfare have been from the fields like genetic engineering; transgenic plants and animals; genomics, proteomics, monoclonal antibodies for the diagnosis of disease, gene therapy etc. Fourteen authoritative chapters written by experts having experience in academics and research on current developments and future trends in biotechnology have been empathized. The book provides a detailed account of various methodologies used in biotechnology i.e. High capacity vectors, DNA sequencing dealing with next generation sequencing, Molecular markers, DNA microarray technology, as well as Proteomics that have revolutionized biotechnology with a wide array of applications. The book not only presents a well-founded explanation of the topics but also aims to present up-to-date reviews of current research efforts, some thoughtful discussions on the potential benefits and risks involved in producing biotechnological products and the challenges of bringing such products to market. It will prove to be an excellent reference work for both academicians and researchers, indicating new starting points to young researchers for new projects in the field. The book is intended for biotechnologist, biologist, researchers, teachers and students of Biosciences and Biotechnology.
Nanotherapeutic Strategies And New Pharmaceuticals Part 2
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Author : Shahid Khan
language : en
Publisher: Bentham Science Publishers
Release Date : 2021-12-07
Nanotherapeutic Strategies And New Pharmaceuticals Part 2 written by Shahid Khan and has been published by Bentham Science Publishers this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-12-07 with Medical categories.
Advances in technology have enabled medicinal chemists to discover and formulate several highly specific, biocompatible, and non-toxic therapeutic agents for clinical applications. Nanotechnology has achieved significant progress in the last few decades and is crucial in every field of science and technology. Nanotechnology-based pharmaceuticals offer multifaceted and alternative methodologies in comparison to the limitations of many conventional clinical therapies. Expertise in designing and developing nanoformulations has helped in targeted drug delivery. Recently, the use of innovative therapeutic agents, particularly in nanomedicine, has accounted for a significant portion of the global pharmaceutical market and is predicted to continue to grow rapidly in the near future. Nanotherapeutic Strategies and New Pharmaceuticals is an accessible multi-part reference which informs the reader about several new techniques based on nanotechnology. The chapters explain relevant topics in detail. The book is designed to encourage and help undergraduate, graduate and post-graduate students in the field of nanotherapeutics, pharmaceuticals and bio-organic chemistry through the use of didactic language and simple illustrations. Part 2 of this book covers the potential of nanotherapeutics and natural therapies for treating neurological diseases, targeting ion channels, signal transduction therapy, gene therapy of single gene mutation diseases and for nanoformulations for special purposes such as wound healing and stimuli-responsive drug delivery. The book also features a chapter that summarizes the types of nanoparticles tailored for specific molecular targets that mediate different diseases. The book set serves as a textbook for students in pharmacology and medical biochemistry, as well as a quick reference for researchers on bio-organic chemistry, as well as general readers interested in nanomedicine.
Psychological Support By Cognitive Behavioral Therapy For Leigh Syndrome
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Author : Edenilson Brandl
language : en
Publisher: Edenilson Brandl
Release Date :
Psychological Support By Cognitive Behavioral Therapy For Leigh Syndrome written by Edenilson Brandl and has been published by Edenilson Brandl this book supported file pdf, txt, epub, kindle and other format this book has been release on with Psychology categories.
Leigh Syndrome is a rare and complex genetic disorder that profoundly affects individuals and their families. As a progressive neurodegenerative condition, it presents numerous challenges that extend beyond physical symptoms, impacting emotional well-being and mental health. Navigating the uncertainties associated with Leigh Syndrome requires a multifaceted approach that encompasses not only medical intervention but also psychological support. In writing this book, my goal is to bridge the gap between the scientific understanding of Leigh Syndrome and the psychological frameworks that can aid in managing its effects. By integrating knowledge from genetics, psychology, and therapy, I aim to provide a comprehensive resource that empowers patients, families, and healthcare professionals alike. Cognitive Behavioral Therapy (CBT) emerges as a powerful tool in this journey. CBT offers practical strategies for coping with the emotional burdens of living with a chronic illness, addressing trauma, and fostering resilience. This book delves into various aspects of CBT, providing a rich array of techniques and tools tailored to the unique experiences of those affected by Leigh Syndrome. Each chapter is designed to build upon the last, guiding the reader through the complexities of genetic illness while promoting mental health and emotional well-being. Throughout this book, I have drawn upon my experiences, research, and insights from individuals who have faced the realities of Leigh Syndrome. Their stories serve as a poignant reminder of the resilience of the human spirit and the critical importance of psychological support. It is my hope that this work not only informs but also inspires hope and healing. I invite you to explore the content within these pages, whether you are a patient, a caregiver, a healthcare professional, or simply someone seeking to understand the intricacies of Leigh Syndrome and the potential of cognitive behavioral therapy. Together, we can navigate this challenging landscape, fostering a supportive environment for those affected by this condition.
Development Of Gene Therapies
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Author : Avery McIntosh
language : en
Publisher: CRC Press
Release Date : 2024-05-23
Development Of Gene Therapies written by Avery McIntosh and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-05-23 with Mathematics categories.
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 . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx Insights into commercial models, access hurdles, and health economics of gene therapies Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective
In Vivo And Ex Vivo Gene Therapy For Inherited And Non Inherited Disorders
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Author : Houria Bachtarzi
language : en
Publisher: BoD – Books on Demand
Release Date : 2019-03-13
In Vivo And Ex Vivo Gene Therapy For Inherited And Non Inherited Disorders written by Houria Bachtarzi and has been published by BoD – Books on Demand this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-03-13 with Medical categories.
Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene therapy has seen a massive transformation from a proof-of-concept approach to a clinical reality culminating in the regulatory approval of state-of-the-art products in the European Union and in the United States. This book captures some of the scientific progresses notably in gene transfer technologies and translational development of in vivo and ex vivo gene therapy interventions in the treatment of a broad range of complex and debilitating non-inherited and inherited disorders such as: human immunodeficiency virus 1 (HIV-1) infection, cancer, cystic fibrosis, hereditary retinopathies, haemophilia B, cardiac diseases, and chronic liver fibrosis.
Fetal Gene Therapy For Fetal Muscular Dystrophy
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Author : Edenilson Brandl
language : en
Publisher: Edenilson Brandl
Release Date : 2025-06-28
Fetal Gene Therapy For Fetal Muscular Dystrophy written by Edenilson Brandl and has been published by Edenilson Brandl this book supported file pdf, txt, epub, kindle and other format this book has been release on 2025-06-28 with Health & Fitness categories.
Gene therapy, one of the most remarkable achievements in science and medicine, has played a crucial role in the search for solutions to debilitating genetic diseases. However, its application in the fetal stage has been a particularly challenging and promising field. This book, "Fetal Gene Therapy for Fetal Muscular Dystrophy," takes a deep dive into the cutting edge of medicine, exploring the advances, complexities, and implications of this exciting scientific journey. Fetal muscular dystrophy is a condition that affects countless families around the world, causing untold suffering and challenges. But as science advances, so does hope. In this book, we will examine how fetal gene therapy has become a promising treatment for this disease and many others. Throughout these pages, we’ll take readers on a fascinating tour of the world of fetal gene therapy, delving into the latest techniques and innovations that are redefining fetal medicine. From the use of viral vectors to nanotechnology and gene editing with CRISPR, we’ll cover the exciting methods being explored to treat and prevent genetic conditions in the fetus. However, fetal gene therapy is not just about science and technology; it also raises profound and thought-provoking questions about ethics, regulation, and equity in access to medical advances. We will explore these sensitive issues, recognizing that every scientific advance carries with it ethical responsibilities and dilemmas. This book is dedicated to all patients and families facing genetic diseases, as well as to the scientists, physicians, and researchers who work tirelessly to create a future where fetal gene therapy can bring hope and cure. Over the following pages, I invite you to delve deeper into this exciting scientific journey, explore the complexities and promises of fetal gene therapy, and consider the profound implications it has for medicine and humanity. Together, we can envision a healthier, more hopeful future for generations to come.
Fetal Gene Therapy For Fetal Phenylketonuria
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Author : Edenilson Brandl
language : en
Publisher: Edenilson Brandl
Release Date : 2025-06-28
Fetal Gene Therapy For Fetal Phenylketonuria written by Edenilson Brandl and has been published by Edenilson Brandl this book supported file pdf, txt, epub, kindle and other format this book has been release on 2025-06-28 with Health & Fitness categories.
It is with great enthusiasm that I present this comprehensive work on the cutting edge of medical science: "Fetal Gene Therapy for Fetal Phenylketonuria". At the heart of this work, we explore the fascinating horizons of genetic intervention in fetal development, particularly aimed at correcting the manifestations of fetal phenylketonuria. Throughout these pages, we navigate the intricate intricacies of messenger RNA (mRNA) injection, viral vectors, and gene-editing technologies such as TALENs and CRISPR-Cas9, revealing innovative strategies for expressing or correcting specific proteins in the fetal environment. The journey includes not only technological advances, but also explores the intersections between advanced fetal medicine, legislation, ethics, and the psychosocial implications of fetal gene therapy. However, we cannot ignore the significant challenges and complexities associated with genetic manipulation at such a crucial stage of human development. Each chapter is meticulously crafted to provide a comprehensive understanding, from an introduction to the fundamentals of fetal gene therapy to future perspectives and potential long-term implications. This book is intended for researchers, healthcare professionals, students, and anyone interested in delving into the frontiers of modern medicine. By sharing these findings and reflections, I hope to contribute to the advancement of knowledge and stimulate dialogue about the promising future and ethical challenges associated with fetal gene therapy. May this work inspire those who seek to understand and shape the future of fetal medicine.
Psychological Support By Cognitive Behavioral Therapy For Hyper Igm Syndrome
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Author : Edenilson Brandl
language : en
Publisher: Edenilson Brandl
Release Date :
Psychological Support By Cognitive Behavioral Therapy For Hyper Igm Syndrome written by Edenilson Brandl and has been published by Edenilson Brandl this book supported file pdf, txt, epub, kindle and other format this book has been release on with Psychology categories.
In recent years, the understanding of genetic disorders has advanced significantly, shedding light on the intricate interplay between our genetic makeup and psychological well-being. Among these conditions, Hyper-IgM Syndrome stands out, not only for its genetic complexities but also for the profound psychological challenges it poses to those affected and their families. This book aims to bridge the gap between genetic knowledge and psychological support, providing a comprehensive resource for individuals navigating the multifaceted journey of living with Hyper-IgM Syndrome. Hyper-IgM Syndrome is characterized by an inability to produce adequate immunoglobulin G (IgG), resulting in increased susceptibility to infections and a host of other health issues. However, the impact of this syndrome extends far beyond the physical realm. The emotional and psychological toll it takes can lead to feelings of isolation, anxiety, depression, and trauma. Recognizing this, I have dedicated this work to exploring how Cognitive Behavioral Therapy (CBT) can serve as an effective therapeutic approach to address these emotional challenges. The structure of this book is designed to guide readers through various dimensions of Hyper-IgM Syndrome, beginning with a foundational understanding of the condition itself. From trauma and genetic concepts to the principles of CBT, each chapter builds upon the last, creating a comprehensive framework for understanding and addressing both the biological and psychological aspects of the syndrome. Throughout this book, you will find practical tools and strategies rooted in CBT that can empower individuals to manage their symptoms, navigate the complexities of their health journey, and foster resilience. Whether you are a patient, a caregiver, a healthcare professional, or someone simply seeking to understand Hyper-IgM Syndrome better, this book is intended to provide valuable insights and actionable strategies. I would like to express my gratitude to the individuals and families who have shared their stories, insights, and experiences with me. Your courage in facing the challenges of Hyper-IgM Syndrome has inspired this work and underscored the importance of a holistic approach to health that encompasses both mind and body. As you embark on this journey through the pages of this book, I encourage you to engage with the material actively. Whether through self-reflection, journaling, or applying the techniques discussed, I hope you find empowerment and support as you navigate the complexities of Hyper-IgM Syndrome and embrace the possibilities of psychological healing. Thank you for joining me on this journey toward understanding and support.
Cancer Nursing
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Author : Connie Yarbro
language : en
Publisher: Jones & Bartlett Learning
Release Date : 2011
Cancer Nursing written by Connie Yarbro and has been published by Jones & Bartlett Learning this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011 with Medical categories.
A new and revised version of this best-selling reference! For over eighteen years, best-selling Cancer Nursing: Principles and Practice has provided oncology nurses with the latest information on new trends in the rapidly changing science of oncology. Now, in its Seventh Edition, Cancer Nursing has been completely revised and updated to reflect key new developments. New topics covered include targeted therapy, hypersensitivity reactions, mucositis, and family and caregiver issues. With 27 new chapters featuring insights from key authors, the Seventh Edition is a must-have resource for every oncology nurse.