Interdisciplinary Approaches to Gene Therapy

Interdisciplinary Approaches to Gene Therapy
Author: Stefan Müller
Publisher: Springer Science & Business Media
Total Pages: 377
Release: 2012-12-06
Genre: Medical
ISBN: 3642608299

Current therapies for most human genetic diseases are inadequate. In response to the need for effective treatments, modern molecular genetics is providing tools for an unprecedented new approach to the treatment of diseases; e.g. the direct manipulation of mutant genes or the input on new therapeutic genes. The treatment of human disease by gene transfer has now moved from the theoretical to the practical realm. With the initiation of clinical trials involving somatic gene therapy in different countries, a critical assessment of the different aspects involved with this new technique is necessary. This volume provides an overview on all these interdisciplinary aspects by some well known experts all over the world.

Oversight and Review of Clinical Gene Transfer Protocols

Oversight and Review of Clinical Gene Transfer Protocols
Author: Institute of Medicine
Publisher: National Academies Press
Total Pages: 78
Release: 2014-03-27
Genre: Medical
ISBN: 030929665X

Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

Advances in Animal Genomics

Advances in Animal Genomics
Author: Sukanta Mondal
Publisher: Academic Press
Total Pages: 340
Release: 2020-11-25
Genre: Science
ISBN: 0128206128

Advances in Animal Genomics provides an outstanding collection of integrated strategies involving traditional and modern - omics (structural, functional, comparative and epigenomics) approaches and genomics-assisted breeding methods which animal biotechnologists can utilize to dissect and decode the molecular and gene regulatory networks involved in the complex quantitative yield and stress tolerance traits in livestock. Written by international experts on animal genomics, this book explores the recent advances in high-throughput, next-generation whole genome and transcriptome sequencing, array-based genotyping, and modern bioinformatics approaches which have enabled to produce huge genomic and transcriptomic resources globally on a genome-wide scale. This book is an important resource for researchers, students, educators and professionals in agriculture, veterinary and biotechnology sciences that enables them to solve problems regarding sustainable development with the help of current innovative biotechnologies. - Integrates basic and advanced concepts of animal biotechnology and presents future developments - Describes current high-throughput next-generation whole genome and transcriptome sequencing, array-based genotyping, and modern bioinformatics approaches for sustainable livestock production - Illustrates integrated strategies to dissect and decode the molecular and gene regulatory networks involved in complex quantitative yield and stress tolerance traits in livestock - Ensures readers will gain a strong grasp of biotechnology for sustainable livestock production with its well-illustrated discussion

Somatic Gene Therapy

Somatic Gene Therapy
Author: P.L. Chang
Publisher: CRC Press
Total Pages: 242
Release: 2018-01-17
Genre: Science
ISBN: 1351093665

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.

Mutation-Specific Gene Editing for Blood Disorders

Mutation-Specific Gene Editing for Blood Disorders
Author: Carsten Werner Lederer
Publisher: Frontiers Media SA
Total Pages: 218
Release: 2021-11-25
Genre: Science
ISBN: 2889716880

Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com.

Engineering Strategies for Regenerative Medicine

Engineering Strategies for Regenerative Medicine
Author: Tiago G. Fernandes
Publisher: Academic Press
Total Pages: 286
Release: 2019-11-14
Genre: Science
ISBN: 0128166703

Engineering Strategies for Regenerative Medicine considers how engineering strategies can be applied to accelerate advances in regenerative medicine. The book provides relevant and up-to-date content on key topics, including the interdisciplinary integration of different aspects of stem cell biology and technology, diverse technologies, and their applications. By providing massive amounts of data on each individual, recent scientific advances are rapidly accelerating medicine. Cellular, molecular and genetic parameters from biological samples combined with clinical information can now provide valuable data to scientists, clinicians and ultimately patients, leading to the development of precision medicine. Equally noteworthy are the contributions of stem cell biology, bioengineering and tissue engineering that unravel the mechanisms of disease, regeneration and development. - Considers how engineering strategies can accelerate novel advances in regenerative medicine - Takes an interdisciplinary approach, integrating different aspects of research, technology and application - Provides up-to-date coverage on this rapidly developing area of medicine - Presents insights from an experienced and cross-disciplinary group of researchers and practitioners with close links to industry

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Regulatory Aspects of Gene Therapy and Cell Therapy Products
Author: Maria Cristina Galli
Publisher: Springer
Total Pages: 235
Release: 2015-09-15
Genre: Medical
ISBN: 3319186183

This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Life with Epidermolysis Bullosa (EB)

Life with Epidermolysis Bullosa (EB)
Author: Jo-David Fine
Publisher: Springer Science & Business Media
Total Pages: 352
Release: 2009-04-02
Genre: Medical
ISBN: 3211792716

Epidermolysis bullosa (EB) is a heterogenous group of genodermatoses characterized by the formation of blisters and erosions on skin and mucous membrans from birth on. The cause are mutations in the genes of structural proteins of the junction between epidermis and dermis. This book deals with the treatment of this skin disease itself and its many extracutaneous complications. There is no previous book which has been focused on the therapy and it will be based on evidence-based data derived from the world ́s largest cohort of inherited EB-patients, the American EB Registry. An important chapter will discuss gene therapy in hereditary EB which has been recently successfully performed within a localized skin site on a single EB patient as a proof-of-principle test. Given its unique collective contents, the monograph will provide the primary source for clinical informations of this oftentimes severe multiorgan disease.

Environmental Epigenetics

Environmental Epigenetics
Author: L. Joseph Su
Publisher: Springer
Total Pages: 327
Release: 2015-05-18
Genre: Medical
ISBN: 1447166787

This book examines the toxicological and health implications of environmental epigenetics and provides knowledge through an interdisciplinary approach. Included in this volume are chapters outlining various environmental risk factors such as phthalates and dietary components, life states such as pregnancy and ageing, hormonal and metabolic considerations and specific disease risks such as cancer cardiovascular diseases and other non-communicable diseases. Environmental Epigenetics imparts integrative knowledge of the science of epigenetics and the issues raised in environmental epidemiology. This book is intended to serve both as a reference compendium on environmental epigenetics for scientists in academia, industry and laboratories and as a textbook for graduate level environmental health courses. Environmental Epigenetics imparts integrative knowledge of the science of epigenetics and the issues raised in environmental epidemiology. This book is intended to serve both as a reference compendium on environmental epigenetics for scientists in academia, industry and laboratories and as a textbook for graduate level environmental health courses.

Gene Drives on the Horizon

Gene Drives on the Horizon
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 231
Release: 2016-08-28
Genre: Science
ISBN: 0309437873

Research on gene drive systems is rapidly advancing. Many proposed applications of gene drive research aim to solve environmental and public health challenges, including the reduction of poverty and the burden of vector-borne diseases, such as malaria and dengue, which disproportionately impact low and middle income countries. However, due to their intrinsic qualities of rapid spread and irreversibility, gene drive systems raise many questions with respect to their safety relative to public and environmental health. Because gene drive systems are designed to alter the environments we share in ways that will be hard to anticipate and impossible to completely roll back, questions about the ethics surrounding use of this research are complex and will require very careful exploration. Gene Drives on the Horizon outlines the state of knowledge relative to the science, ethics, public engagement, and risk assessment as they pertain to research directions of gene drive systems and governance of the research process. This report offers principles for responsible practices of gene drive research and related applications for use by investigators, their institutions, the research funders, and regulators.