Completed notes of the course

Pagina 1

1 LECTURE 1 INTRODUCTION TO THERAPEUTIC PROTEINS BIOLOGICAL MEDICINAL PRODUCTS (BIO – THERAPEUTICS/BIOLOGICS) We talk about whenever there is a living organism (a cell) which is involved in the production of the biological product. Pharmaceutical: word that started with very small organic molecules and kept going with even smaller molecules (antibodies). The future is going toward gene therapies and cellular therapies. In the FDA definition the human source is included in the natural sources. Biotherapeutics: something eventually produced by some organism that we can either extract or guide. PROTEIN STRUCTURE Proteins are extremely large molecules. The primary structure is the simple sequence of the aa. The secondary structure does not involve the entire structure of the protein but only one part. It indicates the region where the aa have organized themselves according to some familiar structures. The 3rd structure is the entire structure. It’s the total arrangement in space. The third structure is the one necessary to have bioactivity! The 1st and 2nd aren't enough in order to have a function. The quaternary structure doesn’t always exist. It’s typical of those proteins that perform a specific activity. 2 RECOMBINANT DNA TECHONOLOGY DNA sequences that code a specific gene of interest. We cannot produce synthetically a protein, but we can produce a cell that can produce a protein by inserting the necessary DNA sequence. We have many organisms that we can use to inject the DNA sequence. They produce the same basic proteins but with some differences (ex. some are highly glycosylated). LIVING ORGANISMS FOR PRODUCING BIOLOGICS To produce biologics we can use humans, for instance by taking proteins from the blood (such as albumin), or we can use genetically modified…