Exploring Novel Clinical Trial Designs for Gene-Based Therapies PDF Download
Are you looking for read ebook online? Search for your book and save it on your Kindle device, PC, phones or tablets. Download Exploring Novel Clinical Trial Designs for Gene-Based Therapies PDF full book. Access full book title Exploring Novel Clinical Trial Designs for Gene-Based Therapies by National Academies of Sciences, Engineering, and Medicine. Download full books in PDF and EPUB format.
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127
Get Book Here
Book Description
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127
Get Book Here
Book Description
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Author: Eve K. Nichols
Publisher: Harvard University Press
ISBN: 9780674414808
Category : Medical
Languages : en
Pages : 268
Get Book Here
Book Description
Nichols explores the potential for gene therapy and identifies those who are candidates for it. Having provided a biomedical background for understanding somatic cell gene therapy, she takes a thoughtful look at complex and sensitive issues surrounding ethical, economic, and policy aspects of manipulating human genes.
Author: Roland W. Herzog
Publisher: World Scientific
ISBN: 9814280917
Category : Medical
Languages : en
Pages : 415
Get Book Here
Book Description
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
Author: Manzoor M. Khan
Publisher: Springer Science & Business Media
ISBN: 0387779760
Category : Medical
Languages : en
Pages : 275
Get Book Here
Book Description
During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.
Author: LeRoy Walters
Publisher:
ISBN: 9780195059557
Category : Medical
Languages : en
Pages : 240
Get Book Here
Book Description
The authors of this absorbing new book describe the science of gene therapy in terms easily accessible to the non-specialist, and focus on the controversial ethical and public policy issues surrounding human interventions in human heredity. After a brief survey of the structure and functions of DNA, genes, and cells, Walters and Palmer discuss three major types of potential genetic intervention: somatic cell gene therapy, germ line gene therapy, and genetic enhancements. They start with the current techniques of gene addition, using non-reproductive (somatic) cells in an effort to cure or treat disease. Next they address the technical problems and moral issues facing attempts to prevent disease through genetically modifying early human embryos or sperm and egg cells. These changes would be passed on to future generations. Chapter 4, in many ways the most original part of this volume, confronts the issue of employing genetic means to improve human abilities and appearance. Depending on the techniques employed, such enhancements could affect not only the individuals receiving the intervention but their offspring as well. Three types of genetic enhancements are considered: physical alterations to improve size, reduce the need for sleep, and decelerate aging; intellectual enhancements of memory and general cognitive ability; and moral enhancements for control of violently aggressive behavior. The authors maintain that genetic modifications should be evaluated individually rather than be condemned in principle or as a group. The final chapter summarizes the public review process that human gene therapy proposals have been undergoing in the United States since 1990. Five appendices, providing technical background information along with a complete list of questions raised in the national public review process, supplement the discussion.
Author: P.L. Chang
Publisher: CRC Press
ISBN: 1351093665
Category : Science
Languages : en
Pages : 242
Get Book Here
Book Description
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.
Author: David T. Curiel
Publisher: Academic Press
ISBN: 0128005106
Category : Science
Languages : en
Pages : 870
Get Book Here
Book Description
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
Author:
Publisher: DIANE Publishing
ISBN: 1428923713
Category :
Languages : en
Pages : 112
Get Book Here
Book Description
Offers information on "Human Gene Therapy" (ISSN 1043-0342), a journal published 18 times a year by Mary Ann Liebert, Inc. that focuses on original investigations into the transfer and expression of genes in mammals, including humans. Provides subscriptions details and outlines submissions requirements for prospective authors.
Author: Daan J. A. Crommelin
Publisher: CRC Press
ISBN: 9780415285018
Category : Medical
Languages : en
Pages : 456
Get Book Here
Book Description
The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.
Author:
Publisher:
ISBN:
Category : Electronic government information
Languages : en
Pages : 120
Get Book Here
Book Description
