Second Generation Cell and Gene Based Therapies Book

Second Generation Cell and Gene Based Therapies


  • Author : Alain Vertes
  • Publisher : Academic Press
  • Release Date : 2020-02-07
  • Genre: Science
  • Pages : 846
  • ISBN 10 : 9780128120330
  • Total Read : 97
  • File Size : 19,6 Mb

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Second Generation Cell and Gene Based Therapies Summary:

Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization "Perspectives" section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies

Second Generation Cell and Gene Based Therapies Book

Second Generation Cell and Gene Based Therapies


  • Author : Alain Vertes
  • Publisher : Academic Press
  • Release Date : 2019-06-15
  • Genre: Science
  • Pages : 500
  • ISBN 10 : 0128120347
  • Total Read : 63
  • File Size : 9,7 Mb

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Second Generation Cell and Gene Based Therapies Summary:

Second-Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalization serves as the only book on the market to bridge basic science, clinical therapy, technology development and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths that will ultimately facilitate clinical delivery of cutting-edge, curative products. Chapters cover hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as enabling technologies, such as gene and genome editing. Additionally, deep dives in product fundamentals, the history of the science, the pathobiology of diseases, the scientific and technological bases, and financing and technology adoption constraints are taken in order to unravel what will shape the cytotherapy industry in 2025 and beyond. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization Perspectives section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies

Cell and Gene Therapies Book

Cell and Gene Therapies


  • Author : Miguel-Angel Perales
  • Publisher : Springer
  • Release Date : 2018-11-27
  • Genre: Medical
  • Pages : 288
  • ISBN 10 : 9783319543680
  • Total Read : 81
  • File Size : 9,6 Mb

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Cell and Gene Therapies Summary:

In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Exploring Novel Clinical Trial Designs for Gene Based Therapies Book

Exploring Novel Clinical Trial Designs for Gene Based Therapies


  • Author : National Academies of Sciences, Engineering, and Medicine
  • Publisher : National Academies Press
  • Release Date : 2020-08-27
  • Genre: Medical
  • Pages : 127
  • ISBN 10 : 9780309672986
  • Total Read : 66
  • File Size : 8,7 Mb

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Exploring Novel Clinical Trial Designs for Gene Based Therapies Summary:

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.

Somatic Gene Therapy Book

Somatic Gene Therapy


  • Author : P.L. Chang
  • Publisher : CRC Press
  • Release Date : 2018-01-17
  • Genre: Science
  • Pages : 313
  • ISBN 10 : 9781351085212
  • Total Read : 90
  • File Size : 6,7 Mb

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Somatic Gene Therapy Summary:

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.

From Research to Reality Book

From Research to Reality


  • Author : The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada
  • Publisher : Council of Canadian Academies
  • Release Date : 2020-11-05
  • Genre: Medical
  • Pages : 122
  • ISBN 10 : 9781926522791
  • Total Read : 95
  • File Size : 12,6 Mb

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From Research to Reality Summary:

From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them.

Regulatory Aspects of Gene Therapy and Cell Therapy Products Book

Regulatory Aspects of Gene Therapy and Cell Therapy Products


  • Author : Maria Cristina Galli
  • Publisher : Springer
  • Release Date : 2015-09-15
  • Genre: Medical
  • Pages : 230
  • ISBN 10 : 9783319186184
  • Total Read : 89
  • File Size : 9,6 Mb

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Regulatory Aspects of Gene Therapy and Cell Therapy Products Summary:

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.

A Guide to Human Gene Therapy Book

A Guide to Human Gene Therapy


  • Author : Roland W. Herzog
  • Publisher : World Scientific
  • Release Date : 2010
  • Genre: Medical
  • Pages : 415
  • ISBN 10 : 9789814280914
  • Total Read : 75
  • File Size : 12,9 Mb

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A Guide to Human Gene Therapy Summary:

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