Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging 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, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. - Provides complete coverage of the basic biology of adenoviruses, as well as their 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

This title explains what gene therapy is, how genes are delivered and how they are targeted. It discusses recent gene therapy trials, future applications and considers the ethical and safety issues surrounding gene therapy. Understanding Gene Therapy is a

Amazing medical breakthroughs are made every day. In the past decades, medical researchers have cured diseases that were once deadly and devised new methods to heal that were once unimaginable. This title follows the development of gene therapy, including the discovery of DNA, groundbreaking discoveries and the doctors who made them, and where the science is heading in the future. Learn how gene therapy works and why future applications of the technology will be controversial. Sidebars, full-color photos, a glossary, and well-placed graphs, charts, and maps, enhance this engaging title. Aligned to Common Core Standards and correlated to state standards. Essential Library is an imprint of Abdo Publishing, a division of ABDO.

One of the recent advances in 21st century medicine is the emergence of gene therapies, drugs that affect the basic biology of genetic disease. The field has seen some notable setbacks in the past, but in recent years has exploded as decades of basic science have been successfully translated into the most complex biologics ever constructed, leading to regulatory approval of several gene therapy products in oncology, hematology, neurology, and ophthalmology indications. These drugs are at the apex of biological manufacturing complexity, and have the potential to be disease modifying or even curative. Evidence-based and innovative quantitative clinical development and lifecycle management strategies will be required as fixtures in the development for these unique drugs in order to reach patients in need. Development of Gene Therapies: Strategic, Scientific, and Regulatory Considerations is an unparalleled summary of the current scientific, statistical, developmental, and regulatory aspects of gene therapies, which is fast becoming a core area of the biopharmaceutical industry. This edited volume provides a systematic description of core development topics in gene therapies through 19 peer-reviewed chapters written by subject matter experts in the field. This edited volume is an invaluable resource for business leaders and investors hoping to understand the scientific principles and strategy of a company they may potentially invest in; the family members of someone affected by a genetic disease who wish to understand better how these therapies work and what they might expect as a treatment for a loved one; academic professionals, who want to learn and teach incoming medical, public health, or business students; and seasoned drug developers, who wish to learn more about the about the cutting edge of biopharmaceutical drug development. Key Features: Provides a thorough background on the scientific, manufacturing, and translational concepts and competencies for gene therapies. Covers important strategic aspects of the gene therapy industry, thereby helping investors, drug developers, and regulators gain a better appreciation of the potential value of gene therapies. Expounds on many existing and emerging state-of-the art scientific and technological advances, as well as ethical, pharmacovigilance, and regulatory considerations for gene therapy product development. Presents several case studies of successful development of gene therapies, including two of the most remarkable FDA-approved gene therapy products: Zolgensma and Luxturna. Provides perspectives and forward-looking statements on the future of gene therapies in neurological, in utero, and ultra-rare indications.

In 1982, a meeting of unusual influence was held at the Banbury Conference Center of Cold Spring Harbor Laboratory. After an early attempt at treating clinical disease with transferred genes had ignited public attention and scientific controversy, a group of distinguished biologists and physicians came together to assess practical progress towards gene therapy and what its future might be. The geneticist Ted Friedmann wrote a narrative account of the participants' contributions to the meeting, ending with a personal discussion of ethical issues raised by genetic technologies. His book, the first on gene therapy, was widely read but has long been unavailable. It has been reprinted with a new introduction entitled Gene Therapy 1994, in which the author reviews the field's technical accomplishments and ethical dilemmas. Now that gene therapy has become part of the medical landscape, this volume is of interest as both a historical document and an assessment of the field's current challenges.

1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne

Dr. Mitchell Gaynor, integrative medicine pioneer, details what we can each do individually to keep our bodies healthy.