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27 Cards in this Set
- Front
- Back
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What are biopharmaceuticals? |
Proteins, peptides, antibodies, and oligonucleotides used as drugs |
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What is the difference between first-generation and second-generation biopharmaceuticals? |
First-generations are mainly copies of endogenous proteins or antibodies produced by recombinant DNA technology, while second-generations are engineered to improve the performance of the protein or antibody |
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What percentage of newly-approved drugs is comprised by biopharmaceuticals? |
25% |
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What is a plasmid? |
A section of circular DNA which can be inserted into or modified to change cellular protein expression |
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What are the intrinsic properties of a drug? |
Potency, specificity, and side effects |
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What is the primary method of delivery for biopharmaceuticals? |
Pulmonary, ocular, nasal, and sublingual delivery |
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Why can a peptide or protein not be delivered orally or via injection? |
Because it will be degraded by the enzymes in digestive tract and veins |
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Why can a peptide or protein not cross the blood brain barrier? |
They are too large |
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What are the main drawbacks of using biopharmaceuticals? |
Cost more to manufacture, yields might be low, often unstable, cannot be delivered orally or intravenously, can't cross the blood/brain barrier |
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How is recombinant DNA technology used to treat disease? |
Used to amplify proteins or peptide hormones that are deficient in diseases (e.g. insulin, growth hormone, factor VIII) |
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What are the primary intracellular targets of biopharmaceuticals? |
Mitochondria, nuclei, cytoplasmic enzymes, and intracellular pathogens |
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How is insulin generally administered? |
Subcutaneous injection (pens) |
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What is the difference between short-acting and long-acting insulin? |
Long acting insulin has been modified to increase its half-life |
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What growth factors can be replaced by biopharmaceuticals? |
Erythropoetin, PDGF, bone morphogenic proteins, interferons |
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What is tPA? |
Tissue plasminogen activator, a serine protease enzyme which converts plasminogen to plasmin, leading to increased thrombus degradation |
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What are monoclonal antobodies? |
An antibody produced by a single clone of cells or cell line and consisting of identical antibody molecules |
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What are the properties of monoclonal antibodies? |
They have highly specific binding sites, they are humanized and can overcome the immune response |
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What are examples of diseases treated by interferons? |
Hepatitis, MS |
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What is gene therapy? |
The replacement of defective genes with normal, healthy genes to prevent, alleviate, or cure disease |
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What types of diseases could be cured directly by gene therapy? |
Monogenic diseases |
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What are monogenic diseases? |
Diseases caused by a mutation in a single gene |
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What are examples of monogenic diseases? |
Cystic fibrosis, sickle-cell disease, Tay-Sachs disease |
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What types of diseases have multiple gene components? |
Cancers, cardiovascular diseases, neurodegenerative diseases |
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What makes llamas useful in the engineering of biopharmaceuticals? |
Llamas have particularly small antibodies which can be tailored for human use |
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What is the most common vector for gene therapies? |
Viral (adenovirus, retrovirus, and adeno-associated virus) |
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What are the non-viral vectors for gene therapies? |
Naked DNA, polymers/liposomes, nanoparticles |
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Why are nucleic acid polymers difficult to get across plasma membranes? |
Because they are quite large |
