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18 Cards in this Set
- Front
- Back
natural product chemistry
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IDing the active ingredient in natural products and making that into medicine
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rational drug design
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IDing a target for drug therapy, then working out a drug from there. usually involves modification of an natural hormone/neurotransmitter (like making antihistamine from histamines)
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how to make recombinant proteins
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- insert desired gene into plasmid or virus vector to make recombinant DNA
- transform plasmid into bacteria/insect/yeast cell - transfect plasmid into human cell - transduce virus vector into human cells - purify the protein made by bacteria/insect/yeast/human cell |
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polycythemia
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- too much erythrocytes = too much red blood cells
= viscus blood = heart failure |
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antibodies
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- proteins that bind specifically to other proteins or molecules
- usually used by body to tag infectious things for destruction - can also be used to tag extra proteins in body for destruction |
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chorionic gonadotrophin hormone
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- The hormone that preg. test antibodies test for to see if your pregers
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monoclonal antibodies
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- antibodies that come from single immortalized mouse cell
- quickly destroyed by our immune sys b/c they are seen as foreign bodies |
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Humanized monoclonal antibodies
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- monoclonal antibodies that are still made in mice, but are altered to look human to immune sys
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Erythropoietin (EPO)
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how it works:
- low O2 in tissues tells Kidney to make EPO - EPO is a glycoprotein hormone that tells bone marrow to make RBC - RBC carries O2 to tissues recombinant protein form is used to treat anemia in chemo patients |
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Herceptin
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- ErbB2 binds to ErbB2 receptors and causes receptor dimerization = promotes cell division. is over expressed in some breast cancers.
- Herceptin is humanized monoclonal antibody that binds ErbB2/HER2/neu receptor and prevents ErbB2/HER2/neu from binding to receptor - has side effects like heart attack, so shouldn't be used by people who don't have ErbB2 over expression |
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Disease that can be treated with Gene Therapy since they are caused by a single gene
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- severe combined immune deficiency (SCID)
- hemophilia - cystic fibrosis - sickle cell - musclar dystrophy |
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severe combined immune deficiency (SCID)
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- bubble body disease
- can be treated with bone marrow transplant (but hard to find doner) or gene therapy |
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viral vectors for gene therapy have:
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- packaged DNA that is long -lived and stable
- mutations added to make it not infectious - helper viruses to help it replicate |
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Lentiviruse
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- a retrovirus used for gene therapy (retro - has RNA that is reverse transcribed into DNA, and then inserted into host DNA)
- advantage: permanent treatment - disadvantage: risk of cancer b/c that DNA could insert anywhere (to turn on oncogene or turn off tumor suppressor) |
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Adenoviruses
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- DNA virus that causes colds/eye infections
- advantage: no integrated into host - disadvantage: treatment has to be taken over and over. This leads to immune response that can kill patient |
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Adeno-associated virus
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advantage:
- integrated into host genome = permanent treatment = no immune response - integrated at specific site = less risk of cancer - infects lots of diff cell types Disadvantage - can only package small amount of DNA Example: Glybera |
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siRNA
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- small sections of RNA that cuts specific sequences in mRNA
- sometimes targets wrong thing - treats Ebola and blindness (due to age related macular degeneration) - This is still just theoretical |
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antisense RNA
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- binds to mRNA and inhibits expression
- example: Fomivirsen/Vitravene |