Therapeutic Enzymes: Function and Clinical Implications
Gebonden Engels 2019 9789811377082Samenvatting
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 volume aims to overview these therapeutic enzymes, focusing in particular on more recently approved enzymes produced by recombinant DNA technology. This volume is composed of four sections. Section 1 provides an overview of the production process and biochemical characterization of therapeutic enzymes, while Section 2 focuses upon the engineering strategies and delivery methods of therapeutic enzymes. Section 3 highlights the clinical applications of approved therapeutic enzymes, including aspects on their structure, indications and mechanisms of action. Together with information on these mechanisms, safety and immunogenicity issues and various adverse events of the recombinant enzymes used for therapy are discussed. Section 4, provides discussion on the prospective and future developments of new therapeutic enzymes. This book is aimed at 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.
Specificaties
Lezersrecensies
Inhoudsopgave
Chapter 2. Upstream processing technologies<br>Ian Marison, Dublin City University, Glasnevin, Dublin 9, Ireland<p></p>
<p>Chapter 3. Downstream processing technologies<br>N. Labrou, Department Biotechnology, University of Athens</p>
<p>Chapter 4. Enzyme Manufacturing Process and Quality Control Evaluations<br>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</p>
<p>Chapter 5. Regulatory guidance in the production of therapeutic enzymes<br>John Geigert, PhD, RAC, BioPharmaceutical Quality Solutions</p>
<p>Chapter 6. Biophysical methods for the characterization of Enzyme Therapeutics<br>Bertrand Raynal Patrick England, CNRS-UMR3528, Institut Pasteur, Department of Structural Biology and Chemistry</p>
<p>Chapter 7. Clinical Development of a therapeutic enzyme<br>Baldo BA, 11 Bent Street, Lindfield, NSW, 2070, Australia</p>
<p>Section 2: Engineering and delivery of therapeutic enzymes</p>
<p>Chapter 8. Chemical modifications of therapeutic proteins<br>Mark D. Distefano, Department of Medicinal Chemistry, University of Minnesota, Minneapolis, MN 55455, USA</p>
<p>Chapter 9. Formulation of therapeutic enzymes<br>Sathy V. Balu-Iyer, Department of Pharmaceutical Sciences, University at Buffalo, The State University of New York, Amherst, NY14260, USA</p>
<p>Chapter 10. Engineering of therapeutic enzymes for improving activity<br>Georgiou, G, University of Texas, Austin</p>
<p>Chapter 11. Engineering of therapeutic enzymes for improving stability<br>Tokuriki N, Michael Smith Laboratories, University of British Columbia, Vancouver, British Columbia, Canada</p>
<p>Chapter 12. Design and engineering of deimmunized therapeutic enzymes<br>Griswold KE, Thayer School of Engineering, Dartmouth, Hanover, NH, United States</p>
<p>Chapter 13. Nanoparticles for delivery of therapeutic enzymes<br>Champion JA, Department of Chemical and Biomolecular Engineering, Georgia Institute of Technology, Atlanta, Georgia</p>
Chapter 14. Targeted delivery of therapeutic enzymes<br>Gianneschi NC, Department of Chemistry and Biochemistry, University of California San Diego, La Jolla, CA, 92093, USA<p></p>
<p>Chapter 15. Oral and inhalable enzyme therapies<br>Khosla C, Division of Biology, California Institute of Technology, Pasadena, California, USA</p>
Chapter 16. Regulatory issues<br>Anurag S. Rathore, Ph.D., DBT Center of Excellence for Biopharmaceutical Technology, Indian Institute of Technology, Delhi, Hauz Khas, New Delhi, 110016, India<p></p>
<p>Section 3: Structure, indications and mechanisms for enzymes approved for human therapy</p>
<p>Chapter 17. Enzyme replacement therapies<br>Harmatz P, 1UCSF Benioff Children's Hospital Oakland, Oakland, California</p>
<p>Chapter 18. Pharmacodynamics and pharmacokinetics issues of enzyme replacement therapy<br>Qi Y, BioMarin Pharmaceutical Inc., 105 Digital Dr, Novato, CA, 94949, USA</p>
<p>Chapter 19. Enzyme therapy for lysosomal storage disorders<br>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</p>
<p>Chapter 20. Enzyme therapy for myocardial infarction<br>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</p>
<p>Chapter 21. Enzyme therapy for chronic gout<br>Shannon JA, Philadelphia College of Osteopathic Medicine, School of Pharmacy, Suwanee, GA, USA</p>
<p>Chapter 22. Enzyme therapy for tumor lysis syndrome<br>Lopez-Olivo MA, Department of General Internal Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA</p>
<p>Chapter 23. Enzyme therapy for collagen-based disorders<br>Kaplan FT, Indiana Hand to Shoulder Center, 8501 Harcourt Rd, Indianapolis, IN 46260 USA</p>
<p>Chapter 24. Enzyme therapy for severe combined immunodeficiency disease<br>Onodera M, Department of Human Genetics, National Research Institute for Child Health and Development, Japan</p>
<p>Chapter 25. Enzyme therapy for detoxification of methotrexate<br>Wang A, The University of Texas MD Anderson Cancer Center, Houston, TX, USA</p>
<p>Chapter 26. Enzyme therapy vitreomacular adhesion<br>Rodríguez-Hurtado FJ, Servicio de Oftalmología, Hospital Virgen de las Nieves, Granada, España</p>
<p>Chapter 27. L-Asparaginase as anticancer agent<br>N. Labrou, Department Biotechnology, University of Athens</p>
<p>Chapter 28. Pancreatic enzyme replacement therapy<br>Berry AJ, University of Virginia Health System, Charlottesville, Virginia</p>
Section 4: Prospective and future therapeutic enzymes<p></p>
<p>Chapter 29. Enzymes in metabolic anticancer therapy<br>Claudia Scotti, Department of Experimental Medicine, Section of General Pathology, University of Pavia, Via Ferrata 1, 27100 Pavia, Italy</p>
<p>Chapter 30. L-Arginase for L-Arg depletion therapy in cancer<br>Feun LG, aSylvester Comprehensive Cancer Center, University of Miami, Miami, Florida</p>
<p>Chapter 31. Methionine L-lyase for L-Met depletion therapy in cancer<br>Cavuoto P, CSIRO Food and Nutritional Sciences, P.O. Box 10041, Adelaide BC, SA 5000, Australia</p>
<p>Chapter 32. Enzyme therapy for celiac sprue<br>Freeman HJ, University of British Columbia, Department of Medicine (Gastroenterology) , Vancouver, BC , Canada</p>
<p>Chapter 33. Acid ceramidase<br>Schuchman EH, Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA</p>
<p>Chapter 34. Lysosomal acid lipase<br>Paton DM, University of Auckland, Auckland, New Zealand</p>
<p>Chapter 35. Alkaline phosphatase and hypophosphatasia<br>Millán JL, Sanford Children's Health Research Center, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, 92037, USA</p>
<p>Chapter 36. Enzybiotics: Antibiotic enzymes as drugs and therapeutics<br>Briers Y, Department of Applied Biosciences, Ghent University, Valentin Vaerwyckweg 1, 9000 Ghent, Belgium</p>
<p>Chapter 37. Anti-inflammatory enzymes<br>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.</p>
<p>Chapter 38. Phenylalanine ammonia lyase for the treatment of phenylketonuria<br>Blau N, University Children's Hospital, Division of Inborn Metabolic Diseases , Im Neuenheimer Feld 669, Heidelberg 69120 , Germany</p>
<p>Chapter 39. Clinical applications of Hyaluronidase<br>Gerber PA, Department of Dermatology, Heinrich-Heine-University Düsseldorf, Düsseldorf, Germany.</p>
<p>Chapter 40. Superoxide dismutase and oxidative stress modulation<br>Carillon J, Nutrition & Métabolisme, UMR 204 NutriPass Prévention des Malnutritions et des Pathologies Associées, Université Montpellier 1-2, Montpellier, France.</p>
<p>Chapter 41. Organophosphate hydrolases as catalytic bioscavengers<br>Masson P, Neuropharmacology Laboratory, Kazan Federal University, 18 Kremlevskaia St., 48000 Kazan, Russian Federation. Electronic address: pym.masson@free.fr. </p>
<p>Chapter 42. Novel therapeutic proteases<br>Daugherty PS, Department of Chemical Engineering, University of California, Santa Barbara, California 93106.</p>
<p>Chapter 43. Genome-editing enzymes<br>Liu, DR., Department of Chemistry & Chemical Biology, Harvard University, Cambridge, Massachusetts, USA</p><p>Chapter 44. Gene therapy for enzyme deficiency: current status and future prospects<br>McIvor RS, Department of Genetics, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USA.</p>
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