ISBN-13: 9789811377112 / Angielski / Miękka / 2020 / 386 str.
ISBN-13: 9789811377112 / Angielski / Miękka / 2020 / 386 str.
Section 1: Production and characterization of therapeutic enzymes
Chapter 1. Introduction to therapeutic enzymes
Fuhrmann G, Leroux JC, Institute of Pharmaceutical Sciences Department of Chemistry and Applied Biosciences, ETH Zurich, Wolfgang-Pauli-Str. 10, HCI H 301, 8093, Zurich, Switzerland
Chapter 3. Downstream processing technologies
N. Labrou, Department Biotechnology, University of Athens
Chapter 4. Enzyme Manufacturing Process and Quality Control Evaluations
Ingrid Markovic, Ph.D., Laboratory of Biochemistry, Division of Therapeutic Proteins, Office of Biotechnology Products, Office of Pharmaceutical Science, Center for Drug Evaluation and Research, FDA
Chapter 5. Regulatory guidance in the production of therapeutic enzymes
John Geigert, PhD, RAC, BioPharmaceutical Quality Solutions
Chapter 6. Biophysical methods for the characterization of Enzyme Therapeutics
Bertrand Raynal Patrick England, CNRS-UMR3528, Institut Pasteur, Department of Structural Biology and Chemistry
Chapter 7. Clinical Development of a therapeutic enzyme
Baldo BA, 11 Bent Street, Lindfield, NSW, 2070, Australia
Section 2: Engineering and delivery of therapeutic enzymes
Chapter 8. Chemical modifications of therapeutic proteins
Mark D. Distefano, Department of Medicinal Chemistry, University of Minnesota, Minneapolis, MN 55455, USA
Chapter 9. Formulation of therapeutic enzymes
Sathy V. Balu-Iyer, Department of Pharmaceutical Sciences, University at Buffalo, The State University of New York, Amherst, NY14260, USA
Chapter 10. Engineering of therapeutic enzymes for improving activity
Georgiou, G, University of Texas, Austin
Chapter 11. Engineering of therapeutic enzymes for improving stability
Tokuriki N, Michael Smith Laboratories, University of British Columbia, Vancouver, British Columbia, Canada
Chapter 12. Design and engineering of deimmunized therapeutic enzymes
Griswold KE, Thayer School of Engineering, Dartmouth, Hanover, NH, United States
Chapter 13. Nanoparticles for delivery of therapeutic enzymes
Champion JA, Department of Chemical and Biomolecular Engineering, Georgia Institute of Technology, Atlanta, Georgia
Chapter 15. Oral and inhalable enzyme therapies
Khosla C, Division of Biology, California Institute of Technology, Pasadena, California, USA
Section 3: Structure, indications and mechanisms for enzymes approved for human therapy
Chapter 17. Enzyme replacement therapies
Harmatz P, 1UCSF Benioff Children's Hospital Oakland, Oakland, California
Chapter 18. Pharmacodynamics and pharmacokinetics issues of enzyme replacement therapy
Qi Y, BioMarin Pharmaceutical Inc., 105 Digital Dr, Novato, CA, 94949, USA
Chapter 19. Enzyme therapy for lysosomal storage disorders
Hollak CE, Department of Internal Medicine, Division of Endocrinology and Metabolism, SPHINX, Amsterdam Lysosome Center, Academic Medical Center, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands
Chapter 20. Enzyme therapy for myocardial infarction
Kunamneni A, Center for Global Health, Department of Internal Medicine, University of New Mexico and New Mexico VA Health Care System, Albuquerque - 87108, New Mexico, USA
Chapter 21. Enzyme therapy for chronic gout
Shannon JA, Philadelphia College of Osteopathic Medicine, School of Pharmacy, Suwanee, GA, USA
Chapter 22. Enzyme therapy for tumor lysis syndrome
Lopez-Olivo MA, Department of General Internal Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA
Chapter 23. Enzyme therapy for collagen-based disorders
Kaplan FT, Indiana Hand to Shoulder Center, 8501 Harcourt Rd, Indianapolis, IN 46260 USA
Chapter 24. Enzyme therapy for severe combined immunodeficiency disease
Onodera M, Department of Human Genetics, National Research Institute for Child Health and Development, Japan
Chapter 25. Enzyme therapy for detoxification of methotrexate
Wang A, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Chapter 26. Enzyme therapy vitreomacular adhesion
Rodríguez-Hurtado FJ, Servicio de Oftalmología, Hospital Virgen de las Nieves, Granada, España
Chapter 27. L-Asparaginase as anticancer agent
N. Labrou, Department Biotechnology, University of Athens
Chapter 28. Pancreatic enzyme replacement therapy
Berry AJ, University of Virginia Health System, Charlottesville, Virginia
Chapter 29. Enzymes in metabolic anticancer therapy
Claudia Scotti, Department of Experimental Medicine, Section of General Pathology, University of Pavia, Via Ferrata 1, 27100 Pavia, Italy
Chapter 30. L-Arginase for L-Arg depletion therapy in cancer
Feun LG, aSylvester Comprehensive Cancer Center, University of Miami, Miami, Florida
Chapter 31. Methionine L-lyase for L-Met depletion therapy in cancer
Cavuoto P, CSIRO Food and Nutritional Sciences, P.O. Box 10041, Adelaide BC, SA 5000, Australia
Chapter 32. Enzyme therapy for celiac sprue
Freeman HJ, University of British Columbia, Department of Medicine (Gastroenterology) , Vancouver, BC , Canada
Chapter 33. Acid ceramidase
Schuchman EH, Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA
Chapter 34. Lysosomal acid lipase
Paton DM, University of Auckland, Auckland, New Zealand
Chapter 35. Alkaline phosphatase and hypophosphatasia
Millán JL, Sanford Children's Health Research Center, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, 92037, USA
Chapter 36. Enzybiotics: Antibiotic enzymes as drugs and therapeutics
Briers Y, Department of Applied Biosciences, Ghent University, Valentin Vaerwyckweg 1, 9000 Ghent, Belgium
Chapter 37. Anti-inflammatory enzymes
Muzykantov VR., Department of Pharmacology and Center for Translational Targeted Therapeutics and Nanomedicine of the Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Chapter 38. Phenylalanine ammonia lyase for the treatment of phenylketonuria
Blau N, University Children's Hospital, Division of Inborn Metabolic Diseases , Im Neuenheimer Feld 669, Heidelberg 69120 , Germany
Chapter 39. Clinical applications of Hyaluronidase
Gerber PA, Department of Dermatology, Heinrich-Heine-University Düsseldorf, Düsseldorf, Germany.
Chapter 40. Superoxide dismutase and oxidative stress modulation
Carillon J, Nutrition & Métabolisme, UMR 204 NutriPass Prévention des Malnutritions et des Pathologies Associées, Université Montpellier 1-2, Montpellier, France.
Chapter 41. Organophosphate hydrolases as catalytic bioscavengers
Masson P, Neuropharmacology Laboratory, Kazan Federal University, 18 Kremlevskaia St., 48000 Kazan, Russian Federation. Electronic address: pym.masson@free.fr.
Chapter 42. Novel therapeutic proteases
Daugherty PS, Department of Chemical Engineering, University of California, Santa Barbara, California 93106.
Chapter 43. Genome-editing enzymes
Liu, DR., Department of Chemistry & Chemical Biology, Harvard University, Cambridge, Massachusetts, USA
Chapter 44. Gene therapy for enzyme deficiency: current status and future prospects
McIvor RS, Department of Genetics, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USA.
This edited work presents studies that clarify several aspects of the development and application of therapeutic enzymes. Therapeutic enzymes exhibit fascinating features and opportunities, and represent a significant and promising subcategory of modern biopharmaceuticals for the treatment of several severe diseases. Research and drug developments efforts and the advancements in biotechnology over the past twenty years have greatly assisted the introduction of efficient and safe enzyme-based therapies for a range of both rare and common disorders. The introduction and regulatory approval of twenty different recombinant enzymes has enabled effective enzyme-replacement therapy.
This book covers mainly three areas of recombinant therapeutic enzymes and their clinical and pharmaceutical technology: (i) overview of the production process and biochemical characterization of therapeutic enzymes, (ii) focuses upon the engineering strategies and delivery methods of therapeutic enzymes, (iii) clinical applications of selected therapeutic enzymes, including aspects on their mechanisms of action and information on safety, immunogenicity issues and various adverse events of the enzymes used for therapy.
The topic of this book is particularly relevant to academics, researchers and students undertaking advanced undergraduate/postgraduate programs in the biopharmaceutical/biotechnology area who wish to gain a comprehensive understanding of enzyme-based therapeutic molecules.
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