Therapeutic Enzymes: Function and Clinical Implications

Description

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. Front Matter ....Pages i-viii Production and Purification of Therapeutic Enzymes (M. Ângela Taipa, Pedro Fernandes, Carla C. C. R. de Carvalho)....Pages 1-24 Production of Therapeutic Enzymes by Lentivirus Transgenesis (María Celeste Rodríguez, Natalia Ceaglio, Sebastián Antuña, María Belén Tardivo, Marina Etcheverrigaray, Claudio Prieto)....Pages 25-54 Quality Control and Downstream Processing of Therapeutic Enzymes (David Gervais)....Pages 55-80 X-Ray Crystallography in Structure-Function Characterization of Therapeutic Enzymes (Anastassios C. Papageorgiou)....Pages 81-103 Application of Fluorescence in Studying Therapeutic Enzymes (Zhaoshuai Wang, Caihong Li, Yinan Wei)....Pages 105-114 Key Physicochemical Characteristics Influencing ADME Properties of Therapeutic Proteins (Xing Jing, Yan Hou, William Hallett, Chandrahas G. Sahajwalla, Ping Ji)....Pages 115-129 Stability of Therapeutic Enzymes: Challenges and Recent Advances (Shubhrima Ghosh, Shahenvaz Alam, Anurag S. Rathore, S. K. Khare)....Pages 131-150 Engineering Strategies for Oral Therapeutic Enzymes to Enhance Their Stability and Activity (Philipp Lapuhs, Gregor Fuhrmann)....Pages 151-172 Enzymes in Metabolic Anticancer Therapy (Maristella Maggi, Claudia Scotti)....Pages 173-199 Alkaline Phosphatase Replacement Therapy (Maria Luisa Bianchi, Silvia Vai)....Pages 201-232 Enzybiotics: Enzyme-Based Antibacterials as Therapeutics (Dorien Dams, Yves Briers)....Pages 233-253 Clinical Applications of Hyaluronidase (Gregor Cornelius Weber, Bettina Alexandra Buhren, Holger Schrumpf, Johannes Wohlrab, Peter Arne Gerber)....Pages 255-277 Alkaline Phosphatase Replacement Therapy for Hypophosphatasia in Development and Practice (S. A. Bowden, B. L. Foster)....Pages 279-322 Rational Use of Pancreatic Enzymes for Pancreatic Insufficiency and Pancreatic Pain (Gyanprakash A. Ketwaroo, David Y. Graham)....Pages 323-343 Fibrinolytic Enzymes for Thrombolytic Therapy (Swaroop S. Kumar, Abdulhameed Sabu)....Pages 345-381 Back Matter ....Pages 383-386