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Protein Therapeutics: Blood Proteins and Enzymes
This lecture covers the composition of blood, including cells, proteins, and salts, as well as the functions and examples of blood proteins. It also delves into blood coagulation, donation, and diseases treated with blood products and enzymes, such as alpha 1-antitrypsin deficiency. The presentation explores the identification of missing plasma factors, coagulation pathways, and the Orphan Drug Act. Examples of enzyme drugs and their production through recombinant DNA technology are discussed, along with case studies on diseases like Gaucher disease and Duchenne muscular dystrophy.