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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

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.

The field of non-viral vector research has rapidly progressed since the publication of the first edition. This new edition is expanded to two separate volumes that contain in-depth discussions of different non-viral approaches, including cationic liposomes and polymers, naked DNA and various physical methods of delivery, as well as a comprehensive coverage of the molecular biological designs of the plasmid DNA for reduced toxicity, prolonged expression and tissue or disease specific genes. New developments such as the toxicity of the non-viral vectors and recent advances in nucleic acid therapeutics are fully covered in these volumes.

The promise of gene therapy can be realized only if workable vectors can be found to deliver therapeutic genes. In Viral Vectors for Gene Therapy: Methods and Protocols, leading researchers from academia and biotechnology describe proven molecular methods for the construction, development, and use of virus vectors for gene transfer and gene therapy. Offering detailed step-by-step instructions to ensure successful results, these experts detail the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Additional chapters demonstrate the use of virus vectors in the brain and central nervous system. Each protocol includes a discussion of the principles involved, numerous charts and tables, ample references, and notes on possible problems, troubleshooting, and alternative procedures. Comprehensive and highly practical, Viral Vectors for Gene Therapy: Methods and Protocols provides not only researchers with the basic tools needed to design targeted gene delivery vectors, but also clinicians with an understanding of how to apply viral vectors to the treatment of genetic disorders.

Adenoviral Vectors for Gene Therapy provides detailed and comprehensive coverage of these important therapeutic agents. The topics covered in this book range from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology and the use of adenoviral vectors in preclinical animal models, to regulatory issues which must be considered prior to the initiation of human clinical gene therapy trials. The broad scope of this unique volume provides the reader with a complete understanding of the development and use of adenoviral vectors.Key Features• Provides complete coverage of basic biology of adenoviruses, as well as the construction, propagation and purification of adenoviral vectors• Introduces common strategies for the development of adenoviral vectors along with cutting-edge methods for their improvement• Demonstrates noninvasive imaging of adenovirus-mediated gene transfer• Discusses utility of adenoviral vectors in animal disease models• Considers Federal Drug Administration regulations for human clinical trials

An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors. Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectorsfurnishes a historical overview of genetic therapy highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications illustrates for new practitioners how each stage of genetic therapy is developed details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.

Genetic manipulation of the adult mammalian nervous system is one of the most exciting areas in contemporary neurobiology. The explosive growth of this field has been facilitated by harnessing the power of viruses to transfer genetic material into mammalian cells.Viral Vectors: Gene Therapy and Neuroscience Applications represents the first comprehensive review of viral vector applications to the nervous system by leaders in virology, molecular neurobiology, neuroanatomy, and developmental neurobiology. It serves both as a source of fundamental information for those newly interested in viral vectors and as a compilation of state-of-the-art technologies and applications for more experienced researchers.This work provides expert background information on viral systems, and the broad range of applications will help readers appreciate the current and future impact of viral vectors in both clinical and basic neuroscience.